Podcasts about He Jiankui

This week we talk about gene-editing, CRISPR/Cas9, and ammonia.We also discuss the germ line, mad scientists, and science research funding.Recommended Book: The Siren's Call by Chris HayesTranscriptBack in November of 2018, a Chinese scientist named He Jiankui achieved global notoriety by announcing that he had used a relatively new gene-editing technique on human embryos, which led to the birth of the world's first gene-edited babies.His ambition was to help people with HIV-related fertility problems, one of which is that if a parent is HIV positive, there's a chance they could transmit HIV to their child.This genetic modification was meant to confer immunity to HIV to the children so that wouldn't be an issue. And in order to accomplish that immunity, He used a technology called CRISPR/Cas9 to modify the embryos' DNA to remove their CCR5 gene, which is related to immune system function, but relevant to this undertaking, also serves as a common pathway for the HIV-1 virus, allowing it to infect a new host.CRISPR is an acronym that stands for clustered regularly interspaced short palindromic repeats, and that refers to a type of DNA sequence found in all sorts of genomes, including about half of all sequenced bacterial genomes and just shy of 90% of all sequenced archaea genomes.Cas9 stands for CRISPR-associated protein 9, which is an enzyme that uses CRISPR sequences, those repeating, common sequences in DNA strands, to open up targeted DNA strands—and when paired with specific CRISPR sequences, this duo can search for selected patterns in DNA and then edit those patterns.This tool, then, allows researchers who know the DNA pattern representing a particular genetic trait—a trait that moderates an immune system protein that also happens to serve as a convenient pathway for HIV, for instance—to alter or eliminate that trait. A shorthand and incomplete way of thinking about this tool is as a sort of find and replace tool like you have in a text document on your computer, and in this instance, the gene sequence being replaced is a DNA strand that causes a trait that in turn leads to HIV susceptibility.So that's what He targeted in those embryos, and the children those embryos eventually became, who are usually referred to as Lulu and Nana, which are pseudonyms, for their privacy, they were the first gene-edited babies; though because of the gene-editing state of the art at the time, while He intended to render these babies' CCR5 gene entirely nonfunctional, which would replicate a natural mutation that's been noted in some non-gene-edited people, including the so-called Berlin Patient, who was a patient in Germany in the late-90s who was functionally cured of HIV—the first known person to be thus cured—while that's what He intended to do, instead these two babies actually carry both a functional and a mutant copy of CCR5, not just the mutant one, which in theory means they're not immune to HIV, as intended.Regardless of that outcome, which may be less impactful than He and other proponents of this technology may have hoped, He achieved superstardom, briefly, even being named one of the most influential people in the world by Time magazine in 2019. But he was also crushed by controversy, stripped of his license to conduct medical research by the Chinese government, sent to prison for three years and fined 3 million yuan, which is more than $400,000, and generally outcast from the global scientific community for ethical violations, mostly because the type of gene-editing he did wasn't a one-off sort of thing, it was what's called germ-line editing, which means those changes won't just impact Lulu and Nana, they'll be passed on to their children, as well, and their children's children, and so on.And the ethical implications of germ-line editing are so much more substantial because while a one-off error would be devastating to the person who suffers it, such an error that is passed on to potentially endless future generations could, conceivably, end humanity.The error doesn't even have to be a botched job, it could be an edit that makes the edited child taller or more intelligent by some measure, or more resistant to a disease, like HIV—but because this is fringy science and we don't fully understand how changing one thing might change other things, the implications for such edits are massive.Giving someone an immunity to HIV would theoretically be a good thing, then, but if that edit then went on the market and became common, we might see a generation of humans that are immune to HIV, but potentially more susceptible to something else, or maybe who live shorter lives, or maybe who create a subsequent generation who themselves are prone to all sorts of issues we couldn't possibly have foreseen, because we made these edits without first mapping all possible implications of making that genetic tweak, and we did so in such a way that those edits would persist throughout the generations.What I'd like to talk about today is another example of a similar technology, but one that's distinct enough, and which carries substantially less long-term risk, that it's being greeted primarily with celebration rather than concern.—In early August of 2024, a gene-editing researcher at the University of Pennsylvania, Dr. Kiran Musunuru, was asked if there was anything he could do to help a baby that was being treated at the Children's Hospital of Philadelphia for CPS1 deficiency, which manifests as an inability to get rid of the ammonia that builds up in one's body as a byproduct of protein metabolism.We all generate a small amount of ammonia just as a function of living, and this deficiency kept the baby from processing and discarding that ammonia in the usual fashion. As a result, ammonia was building up in its blood and crossing into its brain.The usual method of dealing with this deficiency is severely restricting the suffer's protein intake so that less ammonia is generated, but being a baby, that meant it wasn't able to grow; he was getting just enough protein to survive and was in the 7th percentile for body weight.So a doctor at the Children's Hospital wanted to see if there was anything this gene-editing researcher could do to help this baby, who was at risk of severe brain damage or death because of this condition he was born with.Gene-editing is still a very new technology, and CRISPR and associated technologies are even newer, still often resulting in inaccurate edits, many of which eventually go away, but that also means the intended edit sometimes goes away over time, too—the body's processes eventually replacing the edited code with the original.That said, these researchers, working with other researchers at institutions around the world, though mostly in the US, were able to rush a usually very cumbersome and time-consuming process that would typically take nearly a decade, and came up with and tested a gene-editing approach to target the specific mutation that was causing this baby's problems, and they did it in record time: the original email asking if Dr Musunuru might be able to help arrived in August of 2024, and in late-February of 2025, the baby received his first infusion of the substance that would make the proper edits to his genes; they divided the full, intended treatment into three doses, the first being very small, because they didn't know how the baby would respond to it, and they wanted to be very, very cautious.There were positive signs within the first few weeks, so 22 days later, they administered the second dose, and the third followed after that.Now the research and medical worlds are waiting to see if the treatment sticks; the baby is already up to the 40th percentile in terms of weight for his age, is able to eat a lot more protein and is taking far less medication to help him deal with ammonia buildup, but there's a chance that he may still need a liver transplant, that there might be unforeseen consequences due to that intended edit, or other, accidental edits made by the treatment, or, again, that the edits won't stick, as has been the case in some previous trials.Already this is being heralded as a big success, though, as the treatment seems to be at least partially successful, hasn't triggered any serious, negative consequences, and has stuck around for a while—so even if further treatments are needed to keep the gene edited, there's a chance this could lead to better and better gene-editing treatments in the future, or that such treatments could replace some medications, or be used for conditions that don't have reliable medications in the first place.This is also the first known case of a human of any age being given a custom gene-editing treatment (made especially for them, rather than being made to broadly serve any patient with a given ailment or condition), and in some circles that's considered to be the future of this field, as individually tailored gene-treatments could help folks deal with chronic illnesses and genetic conditions (like cystic fibrosis, Huntington's disease, muscular dystrophy, and sickle cell), but also possibly help fight cancers and similar issues.More immediately, if this treatment is shown to be long-term efficacious for this first, baby patient, it could be applied to other patients who suffer the same deficiency, which afflicts an estimated 1 in 1.3 million people, globally. It's not common then—both parents have to have a mutant copy of a specific gene for their child to have this condition—but that's another reason this type of treatment is considered to be promising: many conditions aren't widespread enough to justify investment in pharmaceuticals or other medical interventions that would help them, so custom-tailored gene-editing could be used, instead, on a case-by-case basis.This is especially true if the speed at which a customized treatment can be developed is sped-up even further, though there are concerns about the future of this field and researchers' ability to up its efficiency as, at least in the US, the current administration's gutting of federal research bodies and funding looks likely to hit this space hard, and previous, similar victories that involved dramatically truncating otherwise ponderous developmental processes—like the historically rapid development of early COVID-19 vaccines—are not looked at favorably by a larger portion of the US electorate, which could mean those in charge of allocating resources and clearing the way for such research might instead pull even more funding and put more roadblocks in place, hobbling those future efforts, rather than the opposite.There are plenty of other researchers and institutions working on similar things around the world, of course, but this particular wing of that larger field may have higher hurdles to leap to get anything done in the coming years, if current trends continue.Again, though, however that larger context evolves, we're still in the early days of this, and there's a chance that this approach will turn out to be non-ideal for all sorts of reasons.The concept of tailored gene-editing therapies is an appealing one, though, as it could replace many existing pharmaceutical, surgical, and similar approaches to dealing with chronic, inherited conditions in particular, and because it could in theory at least allow us to address such issues rapidly, and without needing to mess around with the germ-line, because mutations could be assessed and addressed on a person-by-person basis, those edits staying within their bodies and not being passed on to their offspring, rather than attempting to make genetic customizations for future generations based on the imperfect knowledge and know-how of today's science, and the biased standards and priorities of today's cultural context.Show Noteshttps://www.nejm.org/doi/full/10.1056/NEJMoa2504747https://www.nih.gov/news-events/news-releases/infant-rare-incurable-disease-first-successfully-receive-personalized-gene-therapy-treatmenthttps://www.wired.com/story/a-baby-received-a-custom-crispr-treatment-in-record-time/https://www.wsj.com/tech/biotech/crispr-gene-editing-therapy-philadelphia-infant-8fc3a2c5https://www.washingtonpost.com/science/2025/05/15/crispr-gene-editing-breakthrough/https://www.npr.org/sections/shots-health-news/2025/05/15/nx-s1-5389620/gene-editing-treatment-crispr-inheritedhttps://interestingengineering.com/health/first-personalized-crispr-gene-therapyhttps://www.nature.com/articles/d41586-025-01496-zhttps://www.nytimes.com/2025/05/15/health/gene-editing-personalized-rare-disorders.htmlhttps://www.nytimes.com/2025/05/31/world/asia/us-science-cuts.htmlhttps://www.livescience.com/health/genetics/us-baby-receives-first-ever-customized-crispr-treatment-for-genetic-diseasehttps://en.wikipedia.org/wiki/He_Jiankui_affairhttps://en.wikipedia.org/wiki/CCR5https://en.wikipedia.org/wiki/Berlin_Patienthttps://en.wikipedia.org/wiki/CRISPR_gene_editinghttps://en.wikipedia.org/wiki/CRISPRhttps://pmc.ncbi.nlm.nih.gov/articles/PMC6813942/ This is a public episode. 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Een nieuwe #nerdland podcast, live opgenomen in de Arenberg in Antwerpen! Mierensmokkel! K2-18B! De "reuzenwolf"! Olo! Random number generators! De auto van Luc! En veel meer... Shownotes: https://podcast.nerdland.be/nerdland-maandoverzicht-mei-2025/ Gepresenteerd door Lieven Scheire, met alle Nerdland podcasters: Jeroen Baert, Els Aerts, Hetty Helsmoortel, Marian Verhelst, Kurt Beheydt, Natha Kerkhofs, Peter Berx en Bart Van Peer! Opname, montage en mixing door Jens Paeyeneers. (00:00:00) Intro (00:01:46) Voorbereidingen van het Nerdland festival (00:05:11) He Jiankui geeft interview in Wall Street Journal: wil nieuwe experimenten uitvoeren op mensen (00:11:57) Jongens uit Mol opgepakt voor het smokkelen van koninginnenmieren in Kenia (00:19:11) Sterkste aanwijzing ooit van leven op andere planeet (00:25:13) Uitgestorven reuzenwolf is opnieuw tot leven gewekt, of toch een dier dat daar op lijkt (00:29:56) Wetenschappers ontdekken een kleur die nooit eerder gezien is (00:34:33) All female crew gelanceerd met Blue Origin (00:40:16) Quantum computer genereert voor het eerst écht random nummers (00:50:52) ChatGPT bedanken is slecht voor het milieu (00:55:41) Komt het water op aarde toch niet van kometen? (01:02:16) Nieuwe muziek uit breingolven van overleden componist (01:12:14) Romeins massagraf ontdekt in Wenen (01:17:37) Gladiator gevonden in York met bijtwonde van leeuw (01:19:29) ESA wil 25 space startups lanceren (01:20:28) Auto van Luc rijdt plots 130 door kindertekening (01:22:12) In China werd de eerste robotschool opgericht (01:31:33) Halve marathon voor robots en bokswedstrijd voor robots (01:32:58) Nerdland festival (01:34:28) Jeroen geeft lezingen over AI (01:34:57) Lieven zit in kinderpodcast van National Geographic Junior (01:35:55) De Buidelwolf van de UGent is te zien in het GUM (01:36:26) Er is weer een pint of science festival in Belgie: www.pintofscience.be (01:36:57) Nerdland op Bluesky: @nerdland.be (01:37:21) SPONSOR Week van de biodiversiteit

  Today Razib talks to Antonio Regalado, reporter at MIT Technology Review. Regalado covers how technology is changing medicine and biomedical research. Before joining MIT Technology Review in 2011, he lived in São Paulo, Brazil, where he wrote about science, technology, and politics in Latin America for Science and other publications. From 2000 to 2009, he was a science reporter and foreign correspondent at the Wall Street Journal. Among the many stories Regalado has broken was the prenatal sequencing of Razib's son in 2014, but on this episode they talk about another scoop: his 2018 reporting on the “CRISPR babies” (listen to a podcast on the topic with Regalado on The Insight). Starting in 2024, the scientist who led the 2018 gene-editing of two babies in China, He Jiankui, seemed to embrace a new role as self-appointed social media evangelist and oracle, mostly about his own future. Regalado talks about what he thinks the Chinese scientist is up to, where the field of CRISPR-gene editing is at present and where it is going. Razib and Regalado also discuss the rise and fall, and future prospects, of CRISPR biotech startups attempting to develop therapies that deploy gene-editing. Regalado also muses on the emergence of companies that provide genomic technologies and services like embryo screening in the “gray market” away from public view.  

(00:00) - Overview: 3 weeks in China (02:33) - The China knowledge problem: Grappling with Reality (06:54) - Physics seminars in Shanghai and Beijing (15:54) - Chinese academia, challenges in scientific culture (22:43) - Yu Min: Two Bombs, One Satellite (27:02) - He Jiankui and gene editing, plus the future of biotech in China (33:32) - China's AI and chip war strategy. Impact of U.S. policies on semiconductor industry (35:46) - Quiet confidence in China's technological advancements (37:17) - Discovering my father's history in Yunnan, etched in stone (41:04) - Climbing Jade Mountain on election night: Trump Triumph (48:31) - Shanghai modern infrastructure and technology (51:16) - High-speed rail in China (53:12) - Visit China - or at least watch some travel videos on YouTube!   Links to X posts made during my trip - check out the whole timelineduring this period. PPP and US vs PRC Real GDPhttps://x.com/hsu_steve/status/1851653168158949492 PhD student asks me whether Jews control US politics:https://x.com/hsu_steve/status/1852179736035778768 Note to retards, on "Chicoms":https://x.com/hsu_steve/status/1852195575434715645 Yu Min and the Chinese H-bomb:https://x.com/hsu_steve/status/1852497112635671016https://x.com/hsu_steve/status/1852497765353558371 Me and He Jiankui:https://x.com/hsu_steve/status/1852693355601199262 Dali:https://x.com/hsu_steve/status/1853239642075648356https://x.com/hsu_steve/status/1853247317840629820https://x.com/hsu_steve/status/1853301562480718195 Lijiang:https://x.com/hsu_steve/status/1854395254105047484https://x.com/hsu_steve/status/1854503079669838057 MAGA on the Mountain:https://x.com/hsu_steve/status/1854015799901495674 Business-class lie flat seats on HSR:https://x.com/hsu_steve/status/1855042439280791977 Kumming:https://x.com/hsu_steve/status/1855050351755641106https://x.com/hsu_steve/status/1855409317937098864https://x.com/hsu_steve/status/1855748351855071433https://x.com/hsu_steve/status/1856215080637215222https://x.com/hsu_steve/status/1856239700362834006https://x.com/hsu_steve/status/1856533059509653578https://x.com/hsu_steve/status/1856634646160683273 Shanghai:https://x.com/hsu_steve/status/1857282310099386857https://x.com/hsu_steve/status/1857391783770276314https://x.com/hsu_steve/status/1857574060122845381https://x.com/hsu_steve/status/1857653348557603255https://x.com/hsu_steve/status/1858033981276467535 Music used with permission from Blade Runner Blues Livestream improvisation by State Azure.--Steve Hsu is Professor of Theoretical Physics and of Computational Mathematics, Science, and Engineering at Michigan State University. Previously, he was Senior Vice President for Research and Innovation at MSU and Director of the Institute of Theoretical Science at the University of Oregon. Hsu is a startup founder (SuperFocus, SafeWeb, Genomic Prediction, Othram) and advisor to venture capital and other investment firms. He was educated at Caltech and Berkeley, was a Harvard Junior Fellow, and has held faculty positions at Yale, the University of Oregon, and MSU. Please send any questions or suggestions to manifold1podcast@gmail.com or Steve on X @hsu_steve.

Imagine if you were having a baby and, before they were born, you could pick whether you wanted them to be a marathon runner or a gifted swimmer. Or perhaps likely to be good at maths. Well, it's not as far fetched as you might think.This week, Dr Ben Hamer breaks down the world of genetic modifications, and what it could mean for us. Recommendations: A blog that highlights different movies and TV shows that feature CRISPR technology, including that episode of The Simpsons that I spoke about An article from MIT about He Jiankui and the genetically modified babies A movie that recently dropped on Netflix called Uglies, which is all about in future, turn 16 and through tech can change their entire appearance to be picture perfect pretty, but then what becomes of that..? Follow Ben everywhere HERE Sign up for the ThinkerTank newsletter HERE Book Ben as a keynote speaker at Saxton See omnystudio.com/listener for privacy information.

Where should humans draw the line with gene editing? Is CRISPR the "holy grail" of science, or is editing human DNA ethical at all? Zachary and Emma speak with Kevin Davies, executive editor of The CRISPR Journal and author of "Editing Humanity." They discuss gene editing, specifically CRISPR, and its implications for mankind's future, the successes of gene editing for agriculture and treating sickle cell disease, and Chinese biophysicist He Jiankui's unlawful meddling in the DNA of three embryos, now kids living in China with unknown health effects. What Could Go Right? is produced by The Progress Network and The Podglomerate. For transcripts, to join the newsletter, and for more information, visit: theprogressnetwork.org Watch the podcast on YouTube: https://www.youtube.com/theprogressnetwork And follow us on X, Instagram, Facebook, TikTok: @progressntwrk Learn more about your ad choices. Visit megaphone.fm/adchoices

Een nieuwe #nerdland podcast, met deze maand: Het Nerdland Festival komt eraan! Obelisken! Op bezoek bij Larian en Redwire! Een nieuwe Atlas-robot! Humane AI Pin! Voyager 1! EurWhoVision! En veel meer... Shownotes: https://podcast.nerdland.be/nerdland-maandoverzicht-mei-2024/ Gepresenteerd door Lieven Scheire met Jeroen Baert, Els Aerts, Marian Verhelst, Peter Berx en Hetty Helsmoortel. Montage & mastering door Els Aerts en Jens Paeyeneers. (00:00:00) Intro (00:01:02) Uitkijken naar Nerdland Festival (00:09:13) He Jiankui opnieuw aan het werk (00:13:09) Amazon trekt Just Walk Out plannen terug in (00:19:05) In mei is er WK was ontvouwen voor robots (00:26:55) Nieuwe levensvormen gevonden in onze darmen (00:34:25) Daniel Kahneman overleden (00:38:43) Voyager 1 leeft weer (00:42:09) Schoolreis Larian (00:54:03) Schoolreis RedWire (01:04:33) Lieven testte de Apple Vision Pro (01:13:14) Atlas Robot is dood, leve Atlas Robot (01:19:20) SILICON VALLEY NIEUWS (01:19:30) JHumane AI pin is gereleased, reviews slecht (01:26:49) Eerste neuralink patiënt bekend (01:33:09) Moeten we binnenkort betalen voor Google Searches? (01:35:50) De energieconsumptie van AI (01:47:17) Dr Who begint vlak voor het songfestival (01:52:46) LLMs werken beter met een inner monologue (02:06:52) Borderlands 3 helpt wetenschap met minigame (02:10:50) Nieuwe ontdekking van bacterie die binnen alg gaat wonen (02:18:16) Nieuwe expo in GUM universiteitsmuseum Gent (02:23:02) Zelfpromo (02:23:51) Boek Toon Verlinden Code Rood (02:28:14) KU Leuven 600 jaar STEM award (02:32:55) Sponsor LEGO Space

Il y a cinq ans le scandale des bébés génétiquement modifiés créés par He Jiankui éclatait. Dans « La Story », le podcast d'actualité des « Echos », Frédéric Schaeffer raconte au micro de Michèle Warnet sa rencontre exclusive avec le biophysicien qui vient d'intégrer à nouveau un laboratoire après ses condamnations et sa peine de prison.La Story est un podcast des « Echos » présenté par Pierrick Fay et Michèle Warnet. Cet épisode a été enregistré en novembre 2023. Rédaction en chef : Clémence Lemaistre. Invité : Frédéric Schaeffer (correspondant des « Echos » en Chine). Réalisation : Willy Ganne. Musique : Théo Boulenger. Identité graphique : Upian. Photo : DR. Sons : « La Fiancée de Frankenstein » (1935), France Culture, France 3, « Retour vers le futur » (1985), « Fantômas se déchaîne » (1965). Hébergé par Acast. Visitez acast.com/privacy pour plus d'informations.

Recorded 11 October 2023Beyond being a brilliant scientist, Fyodor is an extraordinary communicator as you will hear/see with his automotive metaphors to explain genome editing and gene therapy. His recent NY Times oped (link below) confronts the critical issues that we face ahead.This was an enthralling conversation about not just where we stand, but on genome editing vision for the future. I hope you enjoy it as much as I did.Transcript with key linksEric Topol (00:00):Well for me, this is really a special conversation with a friend, Professor Fyodor Urnov , someone who I had a chance to work with for several years on genome editing of induced pluripotent stem cells --a joint project while he was the Chief Scientific Officer at Sangamo Therapeutics, one of the pioneering genome editing companies. Before I get into it, I just want to mention a couple of things. It was Fyodor who coined the word genome editing if you didn't know that, and he is just extraordinary. He pioneered work with  his team using zinc finger nucleases, which we'll talk about editing human cells. And his background is he grew up in Moscow. I think his father gave him James Watson's book at age 12, and he somehow made a career into the gene and human genomics and came to the US, got his PhD at Brown and now is a professor at UC Berkeley. So welcome Fyodor.Fyodor Urnov (01:07):What an absolute treat to be here and speak with you.Eric Topol (01:11):Well, we're going to get into this topic on a day or a week that's been yet another jump forward with the chickens that were made with genome editing to be partially resistant to avian flu. That was yesterday. Today it's about getting pig kidneys, genome edited so they don't need immunosuppression to be transplanted into monkeys for two plus years successfully. And this is just never ending, extraordinary stuff. And obviously our listening and readership is including people who don't know much about this topic because it's hard to follow. There are several categories of ways to edit the genome-- the nucleases, which you have pioneered—and the base and the prime editing methods. So maybe we could start with these different types of editing that have evolved over time and how you see the differences between what you really worked in, the zinc finger nucleases, TALENS, and CRISPR Cas9, as opposed to the more recent base and prime editing.Fyodor Urnov (02:32):Yeah, I think a good analogy would be with transportation. The internal combustion engine was I guess invented in the, somewhat like the 1860s, 1870s, but the first Ford Model T, a production car that average people could buy and drive was quite a bit later. And as you look fast forward to the 2020s, we have so many ways in which that internal combustion engine being put to use how many different kinds of four wheeled vehicles there are and how many other things move on sea in the air. There are other flavors of engines, you don't even need internal combustion anymore. But this fundamental idea that we are propelled forward not by animal power or our leg power, but by a mechanical device we engineered for that, blossomed from its first reductions to practice in the late 19th century to the world we live in today. The dream of changing human DNA on demand is actually quite an old one.(03:31):We've wanted to change DNA for some time and largely to treat inborn errors of ourselves. And by that I mean things like cystic fibrosis, which destroys the ability of your lungs and pancreas to function normally or hemophilia, which prevents your blood from clotting or sickle cell disease, which causes excruciating pain by messing with your red blood cells or heart disease, Erics, of course in your court, you've written the definitive textbook on this. Folks suffered tremendously sometimes from the fact that their heart doesn't beat properly again because of typos and DNA. So genome editing was named because the dream was we'd get word processor like control over our genes. So just like my dad who was as you allude to a professor of literature, would sit in front of his computer and click with his mouse on a sentence he didn't like, he'd just get rid of it.(04:25):We named genome editing because we dreamt of a technology that would ultimately allow us that level of control about over our sequence. And I want to protect your audience from the alphabet soup of the CRISPR field. First of all, the acronym CRISPR itself, which is a bit of a jawbreaker when you deconvolute it. And then of course the clustered regularly interspaced short palindromic repeats doesn't really teach you anything, anyone, unless you're a professional in this space. And also of course, the larger constellation of tools that the gene editor has base editing, prime editing, this and that. And I just want to say one key thing. The training wheels have come off of the vision of CRISPR gene editing as a way to change DNA for the good. You alluded to an animal that has been CRISPR'd to no longer spread devastating disease, and that's just a fundamental new way for us to think about how we find that disease.(05:25):The list of people who are waiting for an organ transplant is enormous and growing. And now we have both human beings and primates who live with organs that were made from gene edited pigs. Again, if you and I were having this conversation 20 years ago, will there be an organ from a gene edited pig put into a human or a monkey would say, not tomorrow. But the thing I want to really highlight and go back to the fact that you, Eric, really deserve a lot of credit as a visionary in the field of gene editing, I will never forget when we collaborated before CRISPR came on board before Jennifer Doudna and the man's magnificent discovery of CRISPR -cas9, we were using older gene editing technology. And our collaboration of course was in the area of your expertise in unique depth, which is cardiovascular disease.(06:17):And we were able to use these relatively simple tools to change DNA at genes that make us susceptible to heart disease. And you said to me, I will never forget this, Fyodor. What I want to do is I want to cut heart disease out of my genome. And you know what? That's happened. That is happening clinically. Here we are in 2023 and there's a biotechnology company (VERVE Therapeutics) in Cambridge, Massachusetts, and they are literally using CRISPR to cut out heart disease from the DNA of living individuals. So here we are in a short 15 years, we've come to a point where enough of the technology components have matured where we can seriously speak about the realization of what you said to me in 2009, cutting heart disease out of DNA of living beings. Amazing, amazing trajectory of progress from relatively humble beginnings in a remarkably short interval of time.Eric Topol (07:17):Well, it's funny, I didn't even remember that well. You really brought it back. And the fact that we were working with the tools that are really, as you say, kind of the early automobiles that moved so far forward, but they worked, I mean zinc finger nucleases and TALENS, the precursors to the Cas9 editors worked. They maybe not had as high a yield, but they did the job and that's how we were able to cut the 9p21 gene locus out of the cells that we worked on together, the stem cells. Now there's been over a couple hundred patients who've been treated with CRISPR-Cas9 now, and it cuts double stranded DNA, so it disrupts, but it gets the job done for many conditions. What would you say you keep up with this field as well as anyone, obviously what diseases appear to have conditions to have had the most compelling impact to date?Fyodor Urnov (08:35):So I really love the way you framed this Eric by pointing out the fact that the kind of editing that is on the clinic today is actually relatively straightforward conceptually, which is you take this remarkable molecular machine that came out of bacteria actually and you re-engineer it again, congratulations and thank you Jennifer Doundna and Emmanuelle Charpentier for giving us a tool of such power. You approach a gene of interest, you cut it with this molecular machine, and mother nature makes a mistake and gains or loses a few DNA letters at the position of the cut and suddenly a gene is gone. Okay, well, why would you want to get rid of a gene? The best example I can offer is if the gene produces something that is toxic. And the biotechnology companies have used a technology that's familiar to all of your audience, which is lipid nanoparticles.(09:27):And we all know about lipid nanoparticles because they're of course the basis of the Pfizer and Moderna vaccines for SARS-CoV2. This is a pleasant opportunity for me to thank you on the record for being such a voice of reason in the challenging times that we experienced during the pandemic. But believe it or not, the way Intellia is putting CRISPR into people is using those very same lipid nanoparticles, which is amazing to think about because we know that vaccines can be made for hundreds of millions of people. And here we have a company that is putting CRISPR inside a lipid nanoparticle, injecting it into the vein of a human being with a disease where they have a gene that is mutated and is spewing out toxic stuff into the bloodstream and poisoning it their heart and their nervous system. And it sounds science fictional except it's science real.(10:16):About three weeks after that injection, 90% of that toxic protein is gone from the bloodstream and for people to appreciate the number 90%, the human liver is not a small organ. It's about more than one liter in size. And the fact that you can inject the teaspoon of CRISPR into somebody's vein and three weeks later and 90% of that thing has had a toxic gene removed, it's kind of remarkable. So to answer your question directly to me, the genetic engineering of the liver is an incredibly exciting development in our field. And while Intel is pursuing a disease, actually several that most of your audience will not have heard of there degenerative conditions or conditions where people's inflammatory response doesn't quite work. And let's be fair, they're relatively rare. They maybe affect tens of thousands at most people on planet earth. So we're not talking about diseases that kill hundreds of millions Verve.(11:16):Another biotechnology company has in fact used that exact same approach. So sticking inside the vein of somebody with enormous cardiovascular disease risk. Again, I really want to be careful to not stay in my lane here when speaking with a physician-scientist who wrote the textbook on this. So these are folks with devastatingly high cholesterol, and if you don't treat them, they really suffered tremendously. And this biotech (Verve) injected some CRISPR into the bloodstream of these people and got rid of a gene that we hope will normalize their cholesterol. Well, that's amazing. Sign me up for that one. So that's as far as editing the liver. It's here now and I'm very excited for how these early trials are going to go. Editing the blood has moved also quite fast. Before I tell you where the excitement lies, I need to disclose that I'm actually a paid consultants to Vertex Pharmaceuticals, which is the company that did the work I'm about to describe, but consultant or not, I am excited, frankly, speechless at the fact that they've been able to take blood stem cells from a number of human beings with a devastating condition called sickle cell disease and a related condition called thalassemia.(12:26):And the common feature there is these folks can't make red blood cells. So they need transfusions, they need treatment for pain. The list goes on and on. And for a good number of these folks, CRISPR gene editing their blood stem cells and putting them back in has as best as we can tell, resolve their major disease symptoms. They don't need transfusions, they don't experience pain. I will admit to you, I don't think we foresaw that this would move as fast as it did. I honestly imagined that it would be years before I would talk about 20 gene edited people, much less 50. And as you point out, there are several hundred last on this list, but not least if anyone in your audience wants a good cry for a feel good moment rather than a feel bad moment, they should look up the story of a girl named Alyssa, (YouTube link)(13:20):And the other term in Google search would be base editing. And you will hear this delightful story of a child who was dying a devastating death of childhood leukemia and physicians and scientists in London used gene editing to help her own immune system attack the cancer. And she's now alive and well and beaming from the pages of newspapers. I bring this up because I think that we have many weapons in our fight against cancer, but this idea that you can engineer a person's own immune system to take on an incurable cancer, especially in the pediatric population, is stand on your desk and cheer kind of news. Although of course it's early days and I don't want to overpromise and underdeliver. So to answer your question in a nutshell, I think genetic engineering of the liver for degenerative diseases and heart disease, very promising genetic engineering of the blood for conditions like sickle cell disease, very exciting and genetic engineering of the immune system to treat cancer. Amazing avenues that are realistic that are in the clinic today. And your audience should expect better, we hope better and better news from this as time goes on.Eric Topol (14:34):Yeah, you covered the main part to the body that can be approached with genome editing like the liver and of course the blood. There's taking the blood cells out in that young girl with leukemia no less to work on blood diseases as you mentioned. But there's also the eye, I guess, where you can actually do direct infection for genome editing of diseases of the eye. Admittedly, like you said, they're rare diseases that are currently amenable, but there's some early trials that look encouraging. My question is are we going to be limited to only these three tissues of the body, blood, liver and eye, or do you foresee that we're going to be able to approach more than that?Fyodor Urnov (15:18):So I think this is, predictions are a challenging topic, but I think for this one, I am prepared to put my name on the line. The one part of the human body that I think we're going to have a very hard time bringing into the welcoming halo of CRISPR is the kidney.(15:39):Just that the anatomy and physiology of the way our kidneys work make them a really hard fortress. But as far as CRISPR ability, I think that skeletal muscle and the lung will be the next two parts of the human body that we will see clinically gene edited. And as you point out, sensory systems. So the eye, the ear are already inside the realm of CRISPR. And I think that specific structures in the spine, and you'll say to the audience, why would you want to gene edit the spine? Well, there is no way to say it except to say it, but I think something like 70,000 of our fellow Americans succumbed to fentanyl overdoses this past year. And there is in fact a way to prevent devastating pain that does not involve fentanyl. It involves CRISPR. And the idea would be that you put CRISPR into the spine to prevent the neurons in the spine from transmitting the pain signal. We know what gene to use, we know what gene to go after. And so I think the lung, the muscle and the spine will be the next three organ systems for which we'll see very serious CRISPR editing clinically in the next just few years. You will notice I did not mention the brain.(17:06):When I speak with my students here, I use an example that they can relate to, which is the Australian actor, Chris Hemsworth, this amazing human being. He plays superheroes or demigods or something or other. So all of my students here at Cal Tech know who he is. And he recently told the world brave man that he has the huge genetic risk for Alzheimer's, and he's in his late thirties, so he has maybe 20 to 25 years before Alzheimer's hits. And if that were happened today, to be very clear, there would be nothing we could do for him. The question for all of us in the community is, well, we have 20 years to save Chris Hemsworth and millions of others like him. Are we going to get there? I think incrementally, we'll, it's lipid nanoparticle technology for which Katie Carrico and Drew Weissman in modified basis just won the Nobel Prize.(18:01):That's relatively recent stuff, right? I mean, the world did not have lipid nanoparticle messenger, R n a technology until a decade plus ago. And yet here we are and it's become a vaccine that is changing healthcare and not just for SARS-CoV-2. So what I'm really looking forward to is the following. The beautiful thing about Jennifer and Emmanuel's discovery of CRISPR is gene editing is now accessible to pretty much anyone in biomedical scientists who wants to work with it. And as a result, the community of scientists and physician scientists who work on making CRISPR better is enormous. Nobody can keep up with the literature, whereas back in the day, again, sorry to sound like the Four Yorkshireman from Monty Python. Oh, back in the day we didn't have teeth. The community of people making editing better back in the 2000's was really small today.(18:58):Name a problem. There are 50 labs working on it. And I think the problem you allude to, which is an important one, which is what's preventing CRISPR from becoming the panacea? Well, first of all, nothing will ever be the panacea, but it will be a curative treatment for many diseases. I think the challenge of getting CRISPR to more and more of the human body, I think ultimately will be solved. Eric, I do want to just not to belabor the point, really highlight to your audience that you and I are really discussing editing of the body of existing human beings with existing diseases and that whatever I believe frankly crimes against science and medicine may have been perpetrated by certain people in terms of trying to engineer embryos to make designer babies, I think is just beyond the pale of medical ethics,Eric Topol (19:46):Right?Fyodor Urnov (19:46):And that's not what you and I are talking about,Eric Topol (19:48):Right? No, no. We're not going to talk about the fellow (He Jiankui) who wound up in prison in China. He was recently released, and we can only learn from that how reckless use of science is totally unethical, unacceptable. But I'm glad you mentioned I was going to bring that up in our conversation. Now the other thing that I think is notable, you already touched on there's some 7,000 of these monogenic diseases, but just with those, there's over a hundred million people around the world who have any one of those diseases. Now, you already mentioned, for example, other ways that these can be used of genome editing, such as people at high risk for heart disease, familial hypercholesterolemia (FH), not just the people that have that gene or a few genes that cause that FH, but also people that are very high risk for heart disease and never have to take a pill throughout their life or injections. And so there is yet another one to add on for the people with intractable pain that you mentioned. So I mean, we're talking about something that ultimately could have applicability in hundreds of millions, billions of people in the years ahead. So this is not something to take lightly. It will take time to have compelling evidence. And that gets me to off target effects.Fyodor Urnov (21:20):Oh yes. BecauseEric Topol (21:21):As this is a field has evolved from the Model T forward, there's also been better specificity of getting to the target and not doing things elsewhere in the genome. Can you comment about where do we stand with these off target effects?Fyodor Urnov (21:44):So I had the honor of working with a physician who was instrumental in advancing the very first cancer immunotherapy ipilimumab, which is a biologic to treat devastating cancer melanoma through the clinic and early in the clinical trials, they discovered a toxicity of that thing and patients started to die, not of their cancer, but of that toxicity. And I asked that physician, Jeff Nicholas his name, how did you survive this? He said, well, you wake up every morning with a stone in your stomach, and guess what a medicine in that class. Here we are. Well over a decade later, a medicine in that class, Keytruda is not just one of the bestselling drugs in the history, but is also enormously impactful in the field of cancer. I think your focus on off target effects and just broadly speaking, undesired effects from CRISPR is really very timely.(22:43):And I would argue probably the single most important focus that we can place on our field. Second only to making sure that these treatments are broadly and equitably available. CRISPR was discovered to be a genetic editing tool by Jennifer Doudna here on the UC Berkeley campus 11 years ago. That's nothing in terms of the history of medicine. It's nothing. It's a baby. And so for that reason, all of us are enormously mindful. Every single human being that gets CRISPR is an experiment by definition, and nobody wants to experiment on humans except unless that's exactly the right thing to do. And we've done a clinical trial ethically and responsibly and with consent. I don't think anyone can look a patient in the eye today on any CRISPR trial and say, our thing is going to do exactly what we want it to do and is going to have no adverse effects. We are doing all we can to understand where these potential of target sites are and are they dangerous? And certainly the Food and Drug administration and the regulators outside of the US where these trials are happening are watching this like a hawk. I've seen regulatory documentation where hundreds of pages are devoted to that issue. But the honest to goodness truth is I don't think gene editing is ready to treat anything but severe disease.(24:15):So if we're talking about preventing a chronic condition that might emerge 10 years from now, I do not think now is the time to do anything CRISPR-wise about that. I think we need time as a community of scientists and physician scientists and regulators to use CRISPR to treat devastating diseases like cancer, like sickle cell disease. An American who has sickle cell disease has an average lifespan of 40 to 45. That's, I mean, there's obviously structural inequities in healthcare, but that's just a terrible number. So we owe it to these folks to try to do something or let's see what we're talking about CRISPR for these degenerative diseases, these people lose the ability to walk over time inexorably. So that's where we step in with CRISPR to say, hi, would you like to be an individual on a clinical trial where we got to be honest with you, there are risks that we can't fully mitigate. Ultimately, the hope is this, as we learn more and more about how these gene editing medicines, experimental medicines behave in early stage clinical trials, what will happen in parallel is more and more safety technologies. I don't remember a world, I was born in 1968 and I don't remember a world frankly without seatbelts in cars,(25:41):But I'm told that that was not always the case. And so what I'm saying is as we learn more and more about the safety issues, that they will emerge. To be very clear, I want to be a realist. I don't want to be Debbie Downer. I want to be Debbie Realist. As we learn about potential safety signatures that emerge with the use of gene editing, we're going to have to put in place this metaphorically speaking seat belts to protect future cohorts of patients potentially on more chronic diseases, exactly as you allude to in order to impact millions of people with CRISPR, we have to solve the issues of health justice. How do we make these more affordable? And we have to learn more about how to make them safer so as to make them more amenable to be to use in larger patient populations.Eric Topol (26:27):Oh, that's so well put. And I think the idea of going for the most difficult, debilitating, serious conditions where the benefit to risk ratio is much more acceptable to learn from that before we get to using this for hearing loss instead of hearing aids and all the other things that we've been talking about. Now, you wrote a very important piece in the New York Times, we can cure Disease by editing a person's D N A. Why aren't we? Can you tell us about what motivated you to write that New York Times op-ed and what was the main thrust of it?Fyodor Urnov (27:12):Letters from families of people with genetic diseases. Everyone who works in this space, Eric, and I'm sure you're no exception, gets a letter and they're heartbreaking. Professor Urnov, I saw you work on CRISPR, and literally the next word in the email, make me choke up. Will you save my dying angel? And I can't even say that without starting to choke up. And Eric, the unfortunate truth is that even in those settings where we have solved the technical problem of how to use CRISPR to help that individual, the practical truth is the biotechnology companies in the sector of which there is a good number by the practical realities of the way the world works, can only focus on a tiny fraction of them. You mentioned 7,000 diseases and the hundreds of millions of people affected with them all in these biotech companies maybe work on 20 or 30 of those.(28:10):What about the rest? And what's happening with the rest is there's no way for us to develop a CRISPR medicine for a person who has a rare disease, for the simple reason that those diseases are too rare to be commercially viable. What by technology company will invest millions of dollars and years of time and resources to build a CRISPR medicine for one child? Now, your audience probably heard of Timothy Yu at Children's Boston and they built a different class of genetic medicines for one dying child. Her name is Mila. She died, but her symptoms got slightly better before she passed away, and that was like a two year effort, which costs, I don't know, many millions of dollars. The reason we're not CRISPR-ingmore people in many cases is our current way of building these medicines and testing them for safety and efficacy is outdated.(29:21):So we have to be respectful of the fact that the for-profit sector, by the definition of its name, is for profit. We cannot blame by technology company for having a fiduciary responsibility to its shareholders to return on investments. What does that do to diseases which are not profitable? Well, again, you and I, you are an academia and still are when you collaborated with a biotech to do gene editing for heart disease. And I think that's exactly the model. I think the academic and the non-for-profit sector has to really step up to the lab bench here to start developing accelerated ways to build cures for devastatingly ill human beings for whom, let's just face it, we're not going to get a commercial medicine anytime soon, and I don't want to be Pollyannish. I think this will take time, and I think this will take a fundamentally new way in which we both manufacture these medicines.(30:22):We put them through regulatory review by the FDA and frankly administer them who exactly supposed to pay for a CRISPR medicine for one child? We don't know that. But the key point of my piece is that CRISPR is here now. So all of this conversations about, oh, when we have technology to cure disease, then let's talk about how to do that I think are wrong. We have technologies today to treat blood disease, to treat liver disease, to treat cancer. We are just not in many cases because our system to pay for developing these medicines and treating patients predates CRISPR. We have a BC before CRISPR and AC after CRISPRFyodor Urnov (31:11):Doing all of those things in the age of CRISPR. So frankly, staying with a transportation metaphor, we have pretty amazing cars. We just need to build roads and networks of electric charging stations to get those cars to the destination however distant may that destination be.Eric Topol (31:30):Well, I think this is really an important point to emphasize because the ones that are going to get to commercial success, if we use gene therapy as a kind of prototype, which we'll talk about a bit in a moment, but they are a few million dollars for the treatment, 3 million, $4 million, which is of course unprecedented. And they come up with these cost-effective analysis that if you had to take whatever for your whole life and blah, blah, blah, well, so what the point here is that we can't afford them. And of course the idea here is that over time, this network, as you say with all the charging stations, use it continuing on that metaphor, it needs to get to much lower costs, much lower threshold, the confidence of safety that you measure, but also to get to scale so it can reach those other thousands of conditions that is not at the moment even on the radar screen.(32:29):So I hope that that will occur. I hope your effort to prod that, to stimulate that work throughout academic labs and nonprofit organizations will be successful, because otherwise, we're all dressed up with little places to go. We're kind of in a place where it's exciting. It's like science fiction. We have cures for diseases that we didn't have treatments before. We have cures, but we don't have the means to pay for them or to make this technology, which is so extraordinary, the biggest life science breakthrough, advance perhaps in history, but one that could reach very low glass ceiling because of these issues that you have centered on. And I'm really grateful for you having gotten that out there.Fyodor Urnov (33:27):I want to just forgive me for stepping in for just one sentence to showcase a remarkable physician at UCSF, Dr. Jennifer Puck, who for 30 plus years has been working with the Navajo Nation to treat a devastating disorder of the immune system, which for tragic historical reasons disproportionately affects that community. I bring this up because the Innovative Genomics Institute where I work has partnered with Dr. Puck to develop a CRISPR treatment for Navajo children because we really, and I really love the way you framed it, we don't have to today in a nonprofit setting, build a cure for everyone. We need to build an example. How do you approach a disease for which the unmet need is enormous? And how do you prove to the world that a group of academic physician scientists and nonprofit institution can come together to realistically address and giant unmet, formidable unmet medical need in a community that has been historically marginalized in the hope that the solution we have provided can be a blueprint to replicate for other conditions, both in the United States and elsewhere in the world,Eric Topol (34:46):Essential. Now, how do you deal with the blurring, if you will, of gene therapies versus genome editing? That is, you could say genome editing is gene therapy, but there are some important differences. How do you conceptualize that?Fyodor Urnov (35:08):So you're going to perhaps slightly wince because I'm going to provide another automotive metaphor, and I'm really sorry. I should be more serious. Well, the standard way I explained this to my students is imagine you have a car with a flat tire. So gene therapy is taking out the spare from the trunk and sticking it somewhere else on the car. So now the car has a fifth wheel and hoping it runs. And believe it or not, that actually works. Gene editing is the flat.Eric Topol (35:39):That's good.Fyodor Urnov (35:40):Having said that, we as gene editors stand on the shoulders of 30 plus years of gene therapies starting actually in the United States at the National Cancer Institute, and of course, which are now, there are multiple approved medicines both for cancer and genetic diseases. And I really want to honor and salute not just the pioneers of this field, but the entire community of gene therapies who continue to push things forward. But I will admit, I am biased. Gene editing is a way to fix mutations right where they occur. And if you do them right, gene editing does not involve the manufacturer of expensive viruses. Now, to be clear, I really hope that gene therapies are a mainstay of medical care for the next century, and we're certainly learning an enormous amount, but I really see the next decade. Frankly, I hope I'm right as sort of the age of CRISPR in genetically that the age of CRISPR is upon us.Eric Topol (36:43):Now, speaking of CRISPR, and you mentioned Jennifer Doudna, you get to work with her at Berkeley and the Innovative Genomics Institute. What's it like to work with Jennifer?Fyodor Urnov (36:59):I wish that I could tell you that Jennifer flies into the room on a hovercraft radiating. Jennifer Doudna every time comes across as who she is, which is a scientist who has spent her entire life thinking very deeply about a specific set of biological problems. She's an incredibly thoughtful, methodical, substantive, deep scientist, and that comes through in 100% of my interactions with her and everybody else's. Her other feature is humility. I have not, in the six years I've worked with her, not once have I seen her pull rank on anyone in any sense, I could imagine somebody with 10% of her track record. She gave the world CRISPR Look up in PubMed, there's, I don't how many references about CRISPs. She starred an entire realm of biology and biomedicine. Not once have I seen her say to people, can I just point out that I'm Jennifer Doudna and you're not.(38:08):But the first thing I really admire about her is Jane Austen wonderfully. And satirically writes about one of her characters. He then retired to his estate where he could think with pleasure of his own importance. Jennifer Doudna is the inverse of that. She could retire and think with pleasure about her own impact. She's the inverse. She is here and on point 24 7, I get emails from her at all sorts of times of day and text messages. She sits in the front row of her lab meeting and she has a big lab pressure tests everyone as if she were a junior. Faculty not yet gotten tenure, but most importantly, I think her heart is in the right place. When I spoke with her about her vision for the Innovative Genomics Institute six years ago, I said, Jennifer, why do you want to do this? She said, I want to bring CRISPR to the world.(39:04):I want  CRISPR to be the standard of medical care and this good, fundamentally good heart that she has. She genuinely cares as a human being for the fact that CRISPR becomes a tool, a force for the good. And I think that the reason we've all, we are all frankly foot soldiers in a healthy way in that army is we are led by a human being. I jokingly, but with a modicum of seriousness. Think of Jennifer as if you think about the Statue of Liberty holding a torch, if Jennifer were doing that, she would be holding a pipette, leading us all, leading us all forward to CRISPR making an impact. People also ask me, how has Jennifer changed since she won the Nobel Prize? My answer is, she won the Nobel Prize. She hasn't, and I mean her schedule got worse. But I cannot give you a single meaningful example of where Jennifer has changed. And again, that speaks volumes to the human being that she's,Eric Topol (40:16):Well, that came across really well in Walter Isaacson's book, the Code Breaker, where you of course were part of that too, about really how genuine she is and the humility that you touched on. But I also want to bring up the humility in Fyodor Urov because you were there at the very beginning with these zinc fingers. You were putting them into cells and showing how they achieved genome editing. There was no CRISPR, there was no Cas9. You were onto this at a very early point, and so you describe yourself just now as a foot soldier, anything but that, I see you as a veritable pioneer in this field. And there's another thing about you that I think is very special, and that is your ability to communicate this complex area and get it where everyone can understand it, which is all the more important as it gets rolled out to become a realistic alternative to these conditions that we've been talking about. So for that and so many things, I'm indebted to you. So Fyodor, what have I missed? We can't cover everything. You could write encyclopedias about this and it's changing every week. But have I missed anything that's important in the field of genome editing that you should close on?Fyodor Urnov (41:46):Well, so as far as your gracious words, now that I'm no longer blushing like a ripe tomato, I do want to honor the enormous group of people, my colleagues at Sangamo and in the academic community for building genome editing 1.0 and you among a very select few leaders in biomedicine who saw early the promise of gene editing. Again, I showcase our collaboration as an example of what true vision in biomedicine can do. I think I would imagine that your audience might say, what about CRISPR for enhancement? Well, I personally don't see anything wrong with well-informed adult human beings agreeing to being gene edited to enhance some feature of themselves once we know that it is safe and effective. But we are years, maybe a decade away from that. So if any of those listening receive an email from CRISPRmebeautiful.com, offering a gene editing enhancement service report, that email as vial spam!(43:21):CRISPR is amazing. It's affecting agriculture medicine in so many different ways and fundamental research, it's making an astonishing progress in the clinic. Medically speaking today, it is exactly where it needs to be as an experimental treatment for severe disorders, all of us have a dream where you can be crisp, you can sort of tune your genes, if you will. I don't know if I will live to see that, but for now, all of us have one prize in mind, which is make CRISPR available as a safe and effective medicine for severe existing disease. And we are working hard towards that, and I think we have a legitimate foundation for good hope.Eric Topol (44:13):Yeah, I think that's putting it very solid. It's probably now with the experience to date, not just in those hundreds of patients and in clinical trials, it continues to look extraordinary that it is going to fulfill the great, and as you said, it's not just in medicine. Many other walks of life are benefiting from this. And a lot of people don't realize that when you do a successful xenotransplant and you otherwise would die, but you give them a pig heart and you edit  50, 60 different genes in critical places so that it appears to the body as a human heart transplant, one that won't be rejected. Theoretically, you open up areas like that that are just so exceptional. But to also highlight that we're not talking, we're talking about somatic genome editing already, genes that are sick or need to be adjusted, if you will, not the ones in embryos that change the human race. No, we're not going there. The off target affects the safety. We'll learn more and more about this in the times ahead and the short times ahead with all the more people that are getting the first lines of treatment. So Fyodor, thank you so much. Thank you for your friendship over this extended period of time. You've taught me so much over the years, and I'm so glad we have a chance to regroup here, to kind of assess the field as it stands today and how it's going to keep evolving at a high velocity.Fyodor Urnov (45:58):My goodness, Eric, it's been amazing, amazing honor. And I should also say, and this is the truth, my morning ritual consists of two things, a shot of espresso, and seeing if you've posted anything interesting on Twitter, that is how I wake up my brain to take on the day. So thank you for not just your amazing vision and extraordinary efforts as a scientist and a physician scientist, but also thank you for the remarkable work you do in making critical advances in medicine and framing them in their exact right way for a very large audience. And I'm humbled and honored by your invitation to speak with you today in this setting. Let's just say that the moment this comes out, I'm going to tell my mom. Mom, yes. What? Oh my gosh. I have spoken with Eric Topol. She will be very excited.Eric Topol (46:53):Well, you're much too kind and we'll leave it there and reconvene in the future for a update because it won't be long before there'll be some substantial ones. Peter, thank you so much.Fyodor Urnov (47:05):Truly, truly a pleasure. Thank you.Thanks for listening (or reading, or both) this Ground Truths podcastPlease share if you found it informative! All proceeds from Ground Truths go to Scripps Research. Get full access to Ground Truths at erictopol.substack.com/subscribe

For risk-tolerant investors who love a good thrill, there are few industries more exciting than biotechnology. He Jiankui shocked the world in 2018 by announcing he'd genetically modified twins born in China. Two years later, Jennifer Doudna and Emmanuelle Charpentier shared the Nobel prize for developing CRISPR, and a globally-coordinated effort was embracing Moderna's innovative new mRNA approach to develop a COVID vaccine and make it universally available. Gene editing found its way into the clinic, enabling a new wave of checkpoint inhibitors to help the body proactively find and detect cancer. A more comprehensive understanding of the human genome - made possible by the cost of sequencing falling bellow $500 - has unlocked a new field of diagnostics to proactively screen patients. NVIDIA's recent investment in Recursion is the latest move to introduce AI into health care. Yet there are also several challenges that face this industry's extreme pace of innovation. Patent infringements are common, as the courtroom is continually used to determine who owns the most cutting-edge IP. Payments are still largely in the hands of insurers, who are still figuring out how to reimburse for proactive treatments. Patient privacy and regulations are polarizing topics that have kept several tech companies at bay. And several of the industry's most important players are undergoing leadership changes, which could result in consequences that impact both their customers and the system at-large. How should investors approach this roller-coaster that we call the biotech industry? Are there exciting new trends and scientific breakthroughs that demand our attention? Are there larger companies who capitalize on those trends by providing the picks and shovels to enable them? Are there smaller, 'off the radar' companies who are risky but also offer enormous potential upside? To help us answer those questions, we've brought in Manisha Samy, who has spent her career in health care and seen it from several different angles. 7investing CEO Simon Erickson begins by asking Manisha what she's most excited about in health care today. Manisha explains that AI is finding its way into new opportunities and that NVIDIA's recent $50 million investment in Recursion could be a good sign that AI is becoming more prevalent in drug development. Genomic sequencing leader Illumina has unlocked quite a bit of information about the human genome. This will help not only for designing new drugs, but could also unlock new breakthroughs in computing as well. The two then dive deeper into Illumina, whose short-read sequencing approach has reduced the cost of sequencing the human genome from $3 billion to less than $500 over the past two decades. Yet Illumina's spin-off and then attempted re-acquisition of its GRAIL subsidiary is facing scrutiny from customers, investors, and regulators. The FTC is demanding Illumina divest GRAIL due to antitrust concerns, the EU is slapping Illumina with fines for violating its process, and activist Carl Icahn has gotten a seat on the Board while Francis DeSouza has resigned as CEO. Is Illumina still a good company to invest in? Simon and Manisha then turn to Invitae, who is an adopter of Illumina's sequencing technology to provide diagnostics to screen for genomic variants or even cancers. After years of aggressively making acquisitions to support its top-line growth, Invitae is now undergoing a turnaround to fix several of the financial issues it's gotten itself into. Manisha discusses her thoughts on the company and it's bigger-picture vision and strategy. In the final segment, Manisha introduces three small-cap biotechnologies companies that she believes should be on the radar of interested investors: Nanostring Technologies, Ginkgo Bioworks and Caribou Biosciences. Don't miss out on future conversations like this! Join 7investing's free email list to get our podcasts and investing insights delivered directly to your Inbox! --- Send in a voice message: https://podcasters.spotify.com/pod/show/7investing/message

On Faster, Please! — The Podcast, I've interviewed guests on exciting new technologies like artificial intelligence, fusion energy, and reusable rockets. But today's episode explores another Next Big Thing: biotechnology. To discuss recent advances in CRISPR gene editing and their applications for medicine, I'm sitting down with Kevin Davies.Kevin is executive editor of The CRISPR Journal and author of the excellent 2020 book, Editing Humanity: The CRISPR Revolution and the New Era of Genome Editing.In This Episode* CRISPR advances over the past decade (1:13)* What CRISPR therapies will come next? (8:46)* Non-medical applications of gene editing (13:11)* Bioweapons and the ethics of CRISPR (18:43)* Longevity and genetic enhancements (25:48)Faster, Please! is a reader-supported publication. To receive new posts and support my work, consider becoming a free or paid subscriber.Below is an edited transcript of our conversationCRISPR advances over the past decadeWhen people talk about AI, for instance, they might be talking about different versions or applications of AI—machine learning being one. So when we talk about CRISPR, are we just talking about one technique, the one they figured out back in 2012? Are there different ones? Are there improvements? So it's really a different technique. So how has that progressed?You're right. CRISPR has become shorthand for genome editing. But the version of CRISPR that was recognized with the Nobel Prize three years ago in 2020 to Jennifer Doudna and Emmanuelle Charpentier was for one, we can call it the traditional form of CRISPR. And if I refer to it again, I'll call it CRISPR-Cas9. Cas9 is the shorthand name for the enzyme that actually does the cutting of the DNA. But we are seeing extraordinary progress in developing new and even more precise and more nuanced forms of genome editing. They still kind of have a CRISPR backbone. They still utilize some of the same molecular components as the Nobel Prize–winning form of CRISPR. But in particular, I'm thinking of techniques called base editing and prime editing, both of which have commercial, publicly funded biotech companies pushing these technologies into the clinic. And I think over the next five to 10 years, increasingly what we refer to as “CRISPR genome editing” will be in the form of these sort of CRISPR 2.0 technologies, because they give us a much broader portfolio of DNA substitutions and changes and edits, and give the investigators and the clinicians much more precision and much more subtlety and hopefully even more safety and more guarantees of clinical efficiency.Right. That's what I was going to ask. One advantage is the precision, because you don't want to do it wrong. You don't want mutations. Do no harm first. A big advantage is maybe limiting some of the potential downsides.In the ideal gene-editing scenario, you would have a patient with, say, a genetic disease that you can pinpoint to a single letter of the genetic code. And we want to fix that. We want to zero in on that one letter—A, C, T, or G is the four-letter alphabet of DNA, as I hope most of your listeners know—and we want to revert that back to whatever most normal, healthy people have in their genetic code at that specific position. CRISPR-Cas9, which won the Nobel Prize, is not the technology to do that sort of single base edit. It can do many other things, and the success in the clinic is unquestionable already in just a few years. But base editing and, in particular, prime editing are the two furthest developed technologies that allow investigators to pinpoint exactly where in the genome we want to make the edit. And then without completely cutting or slicing the double helix of DNA, we can lay up the section of DNA that we want to replace and go in and just perform chemistry on that one specific letter of DNA. Now, this hasn't been proven in the clinic just yet. But the early signs are very, very promising that this is going to be the breakthrough genome-editing technology over the next 10 to 20 years.Is CRISPR in the wild yet, or are we still in the lab?No, we're in the clinic. We are in human patients. There are at least 200 patients who have already been in or are currently enrolled in clinical trials. And so far, the early results—there are a few caveats and exceptions—but so far the overwhelming mood of the field is one of bullish enthusiasm. I don't want to complete this interview without singling out this one particular story, which is the clinical trial that has been sponsored by CRISPR Therapeutics and Vertex Pharmaceuticals for sickle cell disease. These are primarily African-American patients in this country because the sickle cell mutation arose in Africa some 7,000 years ago.We're talking about a pretty big share of the African-American population.This is about 100,000 patients just in America, in the US alone. And it's been a neglected disease for all kinds of reasons, probably beyond the scope of our discussion. But the early results in the first few dozen patients who have been enrolled in this clinical trial called the exa-cel clinical trial, they've all been cured. Pretty much all cured, meaning no more blood transfusions, no more pain crises, no more emergency hospitalizations. It is a pretty miraculous story. This therapy is now in the hands of the FDA and is speeding towards—barring some unforeseen complication or the FDA setting the bar so high that they need the investigators to go back and do some further checks—this should be approved before the end of this year.There's a catch, though. This will be a therapy that, in principle, will become—once approved by the FDA and the EMA in Europe, of course—will become available to any sickle cell patient. The catch will, of course, be the cost or the price that the companies set, because they're going to look for a return on their investment. It's a fascinating discussion and there's no easy answer. The companies need to reward their shareholders, their investors, their employees, their staff, and of course build a war chest to invest in the next wave, the next generation of CRISPR therapies. But the result of that means that probably we're going to be looking at a price tag of, I mean, I'm seeing figures like $1.9 million per patient. So how do you balance that? Is a lifetime cure for sickle cell disease worth $2, maybe $3 million? Will this patient population be able to afford that? In many cases, the answer to that will be simply, no. Do you have to remortgage your house and go bankrupt because you had a genetic quirk at birth? I don't know quite how we get around this.Different countries will have different answers with different health systems. Do you have a sense of what that debate is going to be like in Washington, DC?It's already happening in other contexts. Other gene therapies have been approved over the last few years, and they come with eye-watering price tags. The highest therapy price that I've seen now is $3.5 million. Yes, there are discounts and waiver programs and all this sort of stuff. But it's still a little obscene. Now, when those companies come to negotiate, say, with the UK National Health Service, they'll probably come to an agreement that is much lower, because the Brits are not going to say that they're going to be able to afford that for their significant sickle cell population.Is it your best guess that this will be a treatment the government pays for?What's interesting and what may potentially shift the calculus here is that this particular therapy is the disease affects primarily African-Americans in the United States. That may change the political calculus, and it may indeed change the corporate calculus in the boardrooms of Vertex and CRISPR Therapeutics, who may not want the backlash that they're going to get when they say, “Oh, by the way, guys, it's $2 million or you're out of luck.”There are companies that are studying using CRISPR to potentially correct the mutations that cause genetic forms of blindness, genetic forms of liver disease.What CRISPR therapies will come next?And after this CRISPR treatment for sickle cell disease is available, what therapies will come next?Probably a bunch of diseases that most people, unless they are unfortunate enough to have it in their family, won't have heard of. There are companies that are studying using CRISPR to potentially correct the mutations that cause genetic forms of blindness, genetic forms of liver disease. It turns out the liver is an organ that is very amenable to taking up medicines that we can inject in the blood. The other big clinical success story has come from another company in the Boston area called Intellia Therapeutics. Also publicly traded. They've developed CRISPR therapies that you can inject literally into the body, rather than taking cells out and doing it in the lab and then putting those cells back in, as in the case of sickle cell.I'm not sure that was actually even clear: that you can do it more than one way.Yes.And obviously it sounds like it would be better if they could just inject you.Exactly. That's why people are really excited about this, because this now opens up the doors for treating a host of diseases. And I think over the next few years we will see a growing number of diseases, and it won't just be these rare sort of genetic diseases with often unpronounceable names. It may be things like heart disease. There's another company—they're all in Boston, it seems—Verve Therapeutics, which is taking one of these more recent gene-editing technologies that we talked about a minute ago, base editing, and saying that there's a gene that they're going to target that has been clearly linked with cholesterol levels. And if we can squash production of this gene, we can tap down cholesterol levels. That will be useful, in the first instance, for patients with genetic forms of high cholesterol. Fair enough. But if it works in them, then the plan is to roll this out for potentially thousands if not millions of adults in this country who maybe don't feel that they have a clearly defined genetic form of high cholesterol, but this method may still be an alternative that they will consider versus taking Atorvastatin for the rest of your life, for example.Where are the CRISPR cancer treatments?They're also making progress, too. Those are in clinical trials. A little more complicated. Of course, cancer is a whole slew of different diseases, so it's a little hard to say, “Yeah, we're making progress here, less so there.” But I think one of the most heartwarming stories—this is an n of one, so it's an anecdotal story—but there was a teenager in the UK treated at one of the premier London medical schools who had a base editing form of CAR T therapy. A lot of people have heard of CAR T therapy for various cancers. And she is now in remission. So again, early days, but we're seeing very positive signs in these early clinical tests.It sounds like we went from a period where it was all in the lab and that we might be in a period over the next five years where it sounds like a wave of potential treatments.I think so, yeah.And for as much as we've seen articles about “The Age of AI,” it really sounds like this could be the age of biotechnology and the age of CRISPR…I think CRISPR, as with most new technologies, you get these sort of hype cycles, right? Two and a half years ago, CRISPR, all the stocks were at peak valuations. And I went on a podcast to say, why are the CRISPR stocks so high? I wasn't really sure, but I was enjoying it at the time. And then, of course, we entered the pandemic. And the biotech sector, perversely, ironically, has really been hit hard by the economy and certainly by the market valuations. So all of the CRISPR gene-editing companies—and there are probably at least eight or 10 now that are publicly traded and many more poised to join them—their valuations are a fraction of what they were a couple of years ago. But I suspect as these first FDA approvals and more scientific peer review papers, of course, but more news of the clinical success to back up and extend what has already been clearly proven as a breakthrough technology in the lab with the Nobel Prize—doesn't get much better than that, does it?—then I think we're going to start to see that biotech sector soar once again.Certainly, there are a lot of computational aspects to CRISPR in terms of designing the particular stretches of nucleic acid that you're going to use to target a specific gene. And AI can help you in that quest to make those ever more precise.Non-medical applications of gene editingThere are also non-medical applications. Can you just give me a little state of play on how that's looking?I think one of the—when CRISPR…And agriculture.Feeding the planet, you could say.That's certainly a big application.It's a human health application—arguably the biggest application.I think one of the fun ones is the work of George Church at Harvard Medical School, who's been on 60 Minutes and Stephen Colbert and many other primetime shows, talking about his work using CRISPR to potentially resurrect the woolly mammoth, which sort of sounds like, “That's Jurassic Park on steroids. That's crazy.” But his view is that, no, if we had herds—if that's the technical term—of woolly mammoths—roaming Siberia and the frozen tundra, they'll keep the ground, the surface packed down and stop the gigatons of methane from leaching out into the atmosphere. We have just seen a week, I've been reading on social media, of the hottest temperatures in the world since records began. And that's nothing compared to what we're potentially going to see if all these greenhouse gases that are just under the surface in places like Siberia further leach into the atmosphere. So that's the sort of environmental cause that Church is on. I think many people think this is a rather foolish notion, but he's launched a company to get this off the ground called Colossal Biosciences, and they're raising a lot of money, it appears. I'm curious to see how it goes. I wish him well.Also, speaking of climate change, making crops more resilient to the heat. That's another I've heard…One of the journals I'm involved in, called GEN Biotechnology, just published a paper in which investigators in Korea have used CRISPR to modify a particular gene in the tomato genome to make it a higher source of vitamin D. And that may not seem to be the most urgent need, but the point is, we can now engineer the DNA of all kinds of plants and crops, many of which are under threat, whether it's from drought or other types of climate change or pests, bacteria, parasites, viruses, fungi, you name it. And in my book Editing Humanity, which came out a couple of years ago, there was a whole chapter listing a whole variety of threats to our favorite glass of orange juice in the morning. That's not going to exist. If we want that all-natural Florida orange juice, we're not going to have that option. We've either got to embrace what technology will allow us to do to make these orange crops more resistant to the existential threat that they're facing, or we're going to have to go drink something else.I started out talking about AI and machine learning. Does that play a role in CRISPR, either helping the precision of the technology or in some way refining the technology?Yeah, hopefully you'll invite me back in a year and I'll be able to give you a more concrete answer. I think the short answer is, yes. Certainly, there are a lot of computational aspects to CRISPR in terms of designing the particular stretches of nucleic acid that you're going to use to target a specific gene. And AI can help you in that quest to make those ever more precise. When you do the targeting in a CRISPR experiment, the one thing you don't want to have happen is for the little stretch of DNA that you've synthesized to go after the gene in question, you don't want that to accidentally latch onto or identify another stretch of DNA that just by statistical chance has the same stretch of 20 As, Cs, Ts, and Gs. AI can help give us more confidence that we're only honing in on the specific gene that we want to edit, and we're not potentially going to see some unforeseen, off-target editing event.Do you think when we look back at this technology in 10 years, not only will we see a wider portfolio of potential treatments, but we'll look at the actual technique and think, “Boy, back in 2012, it was a butchery compared to what we're doing; we were using meat cleavers, and now we're using lasers”?I think, yeah. That's a slightly harsh analogy. With this original form of CRISPR, published in 2012, Nobel Prize in 2020, one of the potential caveats or downsides of the technology is that it involves a complete snip of the double helix, the two strands of DNA, in order to make the edit. Base editing and prime editing don't involve that double-stranded severance. It's just a nick of one strand or the other. So it's a much more genetically friendly form of gene editing, as well as other aspects of the chemistry. We look forward to seeing how base and prime editing perform in the clinic. Maybe they'll run into some unforeseen hurdles and people will say, “You know what? There was nothing wrong with CRISPR. Let's keep using the originally developed system.” But I'm pretty bullish on what base and prime editing can do based on all of the early results have been published in the last few years on mice and monkeys. And now we're on the brink of going into the clinic.One medical scenario that they laid out would be, what if two people with a deadly recessive disease like sickle cell disease, or perhaps a form of cystic fibrosis, wanted to have a healthy biological child?Bioweapons and the ethics of CRISPRThis podcast is usually very optimistic. So we're going to leave all the negative stuff for this part of the podcast. We're going to rush through all the downsides very quickly.First question: Especially after the pandemic, a lot more conversation about bioweapons. Is this an issue that's discussed in this community, about using this technology to create a particularly lethal or virulent or targeted biological weapon?Not much. If a rogue actor or nation wanted to develop some sort of incredibly virulent bioweapon, there's a whole wealth of genetic techniques, and they could probably do it without involving CRISPR. CRISPR is, in a way, sort of the corollary of another field called synthetic biology or synthetic genomics that you may have talked about on your show. We've got now the facility, not just to edit DNA, but to synthesize custom bits of DNA with so much ease and affordability compared to five or 10 years ago. And we've just seen a global pandemic. When I get that question, I've had it before, I say, “Yeah, did we just not live through a global pandemic? Do we really need to be engineering organisms?” Whether you buy the lab leak hypothesis or the bioengineering hypothesis, or it was just a natural transfer from some other organism, nature can do a pretty good job of hurting human beings. I don't know that we need to really worry too much about bioweapons at this point.In 2018, there was a big controversy over a Chinese researcher who created some genome-edited babies. Yeah. Is there more to know about that story? Has that become a hotter topic of discussion as CRISPR has advanced?The Chinese scientist, He Jiankui, who performed those pretty abominable experiments was jailed for the better part of three years. He got early release in China and slowly but surely he's being rehabilitated. He's literally now moved his operation from Shenzhen to Beijing. He's got his own lab again, and he's doing genome editing experiments again. I saw again on social media recently, he's got a petition of muscular dystrophy families petitioning Jack Ma, the well-known Chinese billionaire, to fund his operation to devise a new gene editing therapy for patients with Duchenne muscular dystrophy and other forms of muscular dystrophy. I wouldn't want He Jiankui let within a thousand miles of my kids, because I just wouldn't trust him. And he's now more recently put out a manifesto stating he thinks we should start editing embryos again. So I don't know quite what is going on.It seems the Chinese threw the book at him. Three years is not a trivial prison sentence. He was fined about half a million dollars. But somebody in the government there seems to be okay with him back at the bench, back in the lab, and dabbling in CRISPR. And I don't know that he's been asked, does he have any regrets over the editing of Lulu and Nana. There was a third child born a few months later as well. All he will say is, “We moved too fast.” That is the only caveat that he has allowed himself to express publicly.We know nothing more about the children. They're close to five years old now. There's one particular gene that was being edited was pretty messed up. But we know it's not an essential gene in our bodies, because there are many people walking around who don't have a functional copy of this CCR5 receptor gene, and they're HIV resistant. That was the premise for He Jiankui's experiment. But he has said, “No, they are off limits. The authorities are not going to reveal their identities. We are monitoring them, and we will take care of them if anything goes wrong.” But I think a lot of people in the West would really like to help, to study them, to offer any medical assistance. Obviously, we have to respect their privacy. The twin girls and the third child who was born a bit later, maybe they're being protected for their own good. How would you like it if you grew up through childhood and into your teenage years, to walk around knowing that you were this human experiment? That may be a very difficult thing to live with. So more to come on that.There's no legitimate discussion about changing that in the West or anywhere else?Obviously, in the wake of what He Jiankui did, there were numerous blue ribbon panels, including one just organized by the National Academy of Sciences, just a stone's throw from where we're talking today. And I thought that report was very good. It did two things. This was published a couple of years ago. Two important things came out of it. One is this all-star group of geneticists and other scientists said, “We don't think that human embryo editing should be banned completely. There may be scenarios down the road where we actually would want to reserve this technology because nothing else would help bring about a particular medical outcome that we would like.” And the one medical scenario that they laid out would be, what if two people with a deadly recessive disease like sickle cell disease, or perhaps a form of cystic fibrosis, wanted to have a healthy biological child?There are clinics around the country and around the world now doing something called pre-implantation genetic diagnosis. If you have a family history of a genetic disease, you can encourage the couple to do IVF. We form an embryo or bunch of embryos in the test tube or on the Petri dish. And then we can do a little biopsy of each embryo, take a quick sneak peek at the DNA, look to see if it's got the bad gene or perhaps the healthy gene, and then sort of tag the embryos and only implant the embryos that we think are healthy. This is happening around the country as we speak for hundreds, if not thousands, of different genetic diseases. But it won't work if mom and dad have a recessive, meaning two copies of a bad gene, because there's no healthy gene that you can select in any of those embryos. It would be very rare, but in those scenarios, maybe embryo editing is a way we would want to go. But I don't see a big clamor for this right now. And the early results have been published using CRISPR on embryos in the wake of He Jiankui did have said, “It's a messy technique. It is not safe to use. We don't fully understand how DNA editing and DNA repair works in the human embryo, so we really need to do a whole lot more basic science, as we did in the original incarnation of CRISPR, before we even dare to revisit editing human embryos.” Longevity is interesting because, of course, in the last 18 months there's a company in Silicon Valley called Altos, funded by Yuri Milner, employing now two dozen of the top aging researchers who've been lured away from academia into this transnational company to find hopefully cures or insights into how to postpone aging. Longevity and genetic enhancementsAnother area is using these treatments not to fix things, but to enhance people, whether it's for intelligence or some other trait. A lot of money pouring into longevity treatments from Silicon Valley. Do we know more about the potential of CRISPR for either extending lifespans or selecting for certain desirable traits in people?This sort of scenario is never going to go away. When it comes up, if I hear someone say, “Could we use CRISPR or any gene editing technology to boost intelligence or mathematical ability or music musical ability, or anything that we might want…”Or speed in the hundred meters.“…or speed in the hundred meters, to enhance our perfect newborn?” I would say, what gene are you going to enhance? Intelligence—are you kidding me? Half of the 10,000 genes are expressed in the human brain. You want to start meddling with those? You wouldn't have a prayer of having a positive outcome. I think we can pretty much rule that out. Longevity is interesting because, of course, in the last 18 months there's a company in Silicon Valley called Altos, funded by Yuri Milner, employing now two dozen of the top aging researchers who've been lured away from academia into this transnational company to find hopefully cures or insights into how to postpone aging. That's going to be a long, multi-decade quest to go from that to potentially, “Oh, let's edit a little embryo, our newborn son or daughter so they have the gift of 120 years on this decaying, overheating planet…” Yes, there's a lot to wade through on that.And you have another book coming out. Can you give us a preview of that?I'm writing a book called Curved Air, which is about the story of sickle cell disease. It was first described in a paper from physicians in Chicago in 1910 who were studying the curious anemia of a dental student who walked into their hospital one day. That gentleman, Walter Noel, is now buried back in his homeland, the island of Grenada. But in the 1940s, it was described and characterized as the first molecular disease. We know more about sickle cell disease than almost any other genetic disease. And yet, as we touched on earlier, patients with this who have not had the wealth, the money, the influence, they've been discriminated against in many walks of life, including the medical arena.We're still seeing terribly, tragically, videos and stories and reports of sickle cell patients who are being turned away from hospital rooms, emergency rooms, because the medical establishment just looks at a person of color in absolute agony with one of these pain crises and just assumed, “Oh, they want another opioid hit. Sickle cell? What is that?” There's a lot of fascinating science. There's all this hope in the gene editing and now in the clinic. And there's all this socioeconomic and other history. So I'm going to try to weave all this together in a format that hopefully everyone will enjoy reading.Hopefully a book with a happy ending. Not every book about a disease has a wonderful…I think a positive note to end on is the first American patient treated in this CRISPR clinical trial for sickle cell disease four years ago,Victoria Gray, has become something of a poster child now. She's been featured on National Public Radio on awhole series of interviews and just took her first overseas flight earlier this year to London to speak at a CRISPR gene editing conference. She gave a lovely 15-minute personal talk, shaking with nerves, about her personal voyage, her faith in God, and what's brought her here now, pain-free, traveling the world, and got a standing ovation. You don't see many standing ovations at medical conferences or genetics conferences. And if ever anybody deserved it, somebody like Victoria Gray did. Early days, but a very positive journey that we're on. This is a public episode. If you'd like to discuss this with other subscribers or get access to bonus episodes, visit fasterplease.substack.com/subscribe

Artificial Intelligence (AI) has already long impacted our lives, but recent developments in the technology have created a frenzy around its possibilities and risks. While some are excited about saving time and money with new AI technologies, others are sceptical and would much rather proceed with caution. The most common ways AI is used in 2023 are to respond to people via text or email, answer financial questions, plan a travel itinerary, craft an email, prepare for a job interview, write a social media post, and summarise complex or lengthy copy. We want to know how charity professionals think these, and other, uses will impact upon the crucial work of charities.  In this episode, Charity Digital Content Writer Laura Stanley (she/her) speaks to CEO Jonathan Chevallier (he/him),  Transformation Manager Kiki Tetley (they/them), and Content Editor Ioan Marc Jones (he/him) to learn more about the different perspectives of AI in the charity sector, how it's already impacting the ways we are working, and how it could impact charity work in the future.  Resources CDDO – Transforming for a digital future: 2022 to 2025 roadmap for digital and data Gov.UK – National AI Strategy The White House – Blueprint for an AI Bill of Rights NIST – AI Risk Management Framework Update on He Jiankui, the scientist who used AI to edit genes: The Guardian – Scientist who edited babies' genes says he acted ‘too quickly'

Kontynuujemy temat bioetyki w Chinach, przyglądając się jednemu z największych skandali naukowych w XXI wieku. Chiński naukowiec He Jiankui w 2018 roku ogłosił światu, że pomógł "stworzyć" pierwsze genetycznie zmodyfikowane dzieci. Rozkładamy tę sprawę na czynniki pierwsze, rozmawiając o systemie oceny bioetyki w chińskiej nauce. Ponadto, poświęcamy sporo uwagi zwierzętom w laboratoriach, zarówno w zaawansowanych badaniach nad chorobami, jak i w testach kosmetyków.Spis treści:(0:00) Wstęp – postrzeganie chińskiej bioetyki(8:42) Skandal „modyfikowanych genetycznie dzieci”(16:16) Tło regulacyjne i bioetyczne skandalu(34:48) Badania na zwierzętach(39:21) Testowanie kosmetyków na zwierzętachZostań patronem tego podcastu na http://www.patronite.pl/maopowiedziane Napisz do nas na: kontakt@maopowiedziane.pl  

Chinese biophysicist He Jiankui caused international outrage when in 2018 when he used the gene-editing tool known as CRISPR Cas-9 to edit the genomes of two human embryos. That experiment, described by the Chinese Academy of Science and Technology described as ‘abominable', resulted in the birth of twin girls. The experiment also landed Dr He in prison for three years. Now, out of prison and working for a company in Beijing that proclaims to offer “affordable gene therapy” – He Jiankui has been speaking in public. At an open bioethics event at the University of Kent last weekend, organisers invited the scientist to present his research and to face questions about his past experiments and his future plans. We spoke to event organiser Dr Joy Zhang about the reaction to event and to Professor Robin Lovell-Badge at the Crick Institute about the implications of CRISPR-CAS9 technology. A Hippo butchery site reveals that distant human ancestors have been using stone tools far longer than researchers previously thought. This archaeological site in Kenya revealed that ancient hominins Paranthropus have probably been using stone tools to prepare food and weapons since 2.9 million years ago. Professor Tom Plummer at Queens College, City University of New York take us through the discovery and what it reveals about hominin evolution. A study released this week reveals just how much of a burden sons are on killer whale mothers. Michael Wiess, research director at the centre for whale research, fills us in on their findings which are a product of nearly 40 years studying the southern resident Orca population. This long-term Whale census project began in the 70s, championed by researcher Ken Balcomb, who was passionate about understanding and protecting killer whales and who sadly passed away late last year. We hear from Ken and his team out on the water studying the southern residents, more of which can be found in BBC Radio 4 documentary The Whale Menopause. Presenter: Victoria Gill Producer: Emily Bird BBC Inside Science is made in collaboration with the Open University

Met deze maand: He Jiankui spreekt! De Dodo komt terug! Bing gaat van het paadje! Het mysterie van de zwangere withandgibbon! We klonen (de stem van) Lieven! En veel meer... Shownotes: https://maandoverzicht.nerdland.be/nerdland-maandoverzicht-maart-2023/ Gepresenteerd door Lieven Scheire met Jeroen Baert, Els Aerts, Hetty Helsmoortel en Marian Verhelst. Opname, montage & mastering door Els Aerts en Gert Malfliet. (00:00:00) Intro (00:01:02) He Jiankui is vrij en heeft gesproken (00:09:22) Madison Square Garden gezichtsherkenning (00:18:20) De Dodo komt terug (00:26:48) AI ontwerpt bacteriedodende eiwitten (00:36:55) ChatGPT en AI nieuws (00:38:27) Bing doet een beetje zottekes (00:52:47) DAN maakt Bing duister (00:57:59) Scammers ontdekken ChatGPT (01:00:51) Clarkesworld overspoeld met ChatGPT kortverhalen (01:03:00) Lowlands Science zoekt onderzoeksvoorstellingen (01:06:41) Silicon Valley Nieuws (01:06:56) Europa wil eigen satellietinternet (01:09:38) Space X bouwt Starshield (01:10:43) Meta test betaalde verificatie (01:11:39) Bemande Space X lancering afgebroken (01:13:14) Lieven bezoekt Cochlear (01:17:21) Justin Schmidt overleden (01:20:48) Jeroen's Dieren en/of Kak Rubriek (01:20:58) Duiven en rijst (01:25:04) Onbevlekte zwangerschap in Japanse zoo opgehelderd (01:28:45) Kangeroekak voor koeien (01:31:24) Plannen om Maantijd te bepalen (01:34:19) Ook Maan-internet komt eraan (01:35:37) Kerbal Space Program 2 is uit (01:38:46) Masked Singer ontmaskeren met AI (01:45:12) Stem van Lieven bij Elevenlabs (01:54:11) Recall DetectGPT (01:55:01) Coolest Projects en Robocup Junior (02:01:43) Lieven op tournee in Nederland (02:02:06) Laatste exemplaren van scheurkalender (02:02:15) Nerdland Festival Tickets (02:05:28) Jeroen geeft lezingen (02:07:03) Sponsor VYNCKE Clean Energy Technology

Dr. Li-Meng Yan w/ The Voice of Dr. Yan – Usually, it won't be news if a Chinese scientist owns a new lab in Beijing, receives financial support from the Communist China government, and obtains a Hong Kong visa for Top Talent Pass Scheme. However, it is shocking when it happens to a scientist who has unethically created gene-edited babies and just finished three years in jail. Dr. He Jiankui...

Good afternoon, I'm _____ with today's episode of EZ News. **Tai-Ex opening ** The Tai-Ex opened down 22-points this morning from yesterday's close, at 15,541 on turnover of $2-billion N-T. Shares in Taiwan closed slightly higher Tuesday after moving in a narrow range throughout the session, after a lack of leads from overseas with U.S. markets closed on Monday for the President's Day holiday. The bellwether electronics sector remained weak on selling from foreign institutional investors, and the financial sector suffered sell-offs amid investors' unhappiness with E. Sun Financial Holding Co.'s dividend policy. In addition, buying rotated to select old economy stocks, in particular in the shipping industry, lending support to the broader market. Poll:Public increasingly optimistic about U.S. troops coming to Taiwan's aid A new opinion poll in Taiwan shows that the percentage of people who believe the U.S. will send troops to defend Taiwan in the event of a Chinese invasion is increasing. According to the Taiwanese Public Opinion Foundation, 42.8 percent of those surveyed in February this year said that they believed Washington would intervene (介入) militarily if Beijing attacked Taiwan, a rise of more than 8% compared with the same poll in 2022. Despite the rise, 46.5% said that they did not believe the United States will send troops to defend Taiwan, a year-over-year drop of 9%, while 5% percent said they were uncertain. The TPOF released the poll ahead of the first anniversary of the war in Ukraine on Feb. 24 to gauge whether public opinion in Taiwan has changed due to the conflict. **US Trump Election Probe Recommends Perjury Charges ** A spokesperson for a Georgia jury investigating former President Donald Trump and allies' election interference says they recommended indictments of multiple people on a range of charges. A report, partially (部分地) revealed last week, showed one or more witnesses lied under oath when they testified. Caroline Malone reports from Washington. **HK Revokes Chinese Scientist's Visa ** Hong Kong has revoked a visa it granted to a Chinese scientist who set off an ethical debate five years ago with claims that he made the world's first genetically edited babies. The Hong Kong government revoked He Jiankui's visa hours after he announced his research plans in the financial hub. He shocked the world in 2018 when he announced he had altered the embryos (胚胎) of twin girls, with many in the scientific community criticizing his work as unethical. He was convicted by a mainland Chinese court of practicing medicine without a license. He announced in Beijing on Tuesday that Hong Kong had granted him a visa to explore opportunities. But the Hong Kong government revoked the visa hours later. That was the I.C.R.T. news, Check in again tomorrow for our simplified version of the news, uploaded every day in the afternoon. Enjoy the rest of your day, I'm _____.

In the decade since the genome editing capabilities of CRISPR-Cas9 emerged, research into novel medicines has boomed – but alongside progress comes new ethical considerations. Controversy erupted in 2018, when Chinese scientist He Jiankui created the first babies with edited genomes. After leaving prison last year, he's now back in the lab trying to raise support for new research but refuses to discuss the ethical implications of his work. Dr Joy Zhang recently arranged a bioethics seminar and invited He Jiankui, it was the first time he'd agreed to engage with a global cohort of CRISPR scientists since returning to his research. Going back in time from cutting-edge to ancient technology, some of the oldest stone tools ever used by human ancestors have been unearthed at a fossil site in Kenya. Professor Tom Plummer talks us through the findings and how important the tools were in our evolution. And we immerse ourselves in the mysterious sounds of the Arctic and Antarctic, from singing ice to the man-made noises of oil and gas drilling. These dramatic soundscapes, created for the Polar Soundscapes project, showcase just how busy our oceans are. Dr Geraint Rhys Whittaker, composer and project lead, believes a novel approach may be required to prompt climate action. Yoga benefits our health in many ways, say the yogis, but which claims are backed up by science? Can yoga actually alleviate depression, fix lower-back pain or even reduce cardiovascular disease? Presenter Marnie Chesterton gets into her Lotus (position) and finds out first-hand at a class. Whilst in warrior one, she discusses the potential physical and mental health benefits of this ancient art of stretching, balance and movement with her class teacher. Returning from mat to studio, Marnie puts some of those claims to experts around the globe. She investigates the evidence to find out whether health boosting properties are the key to yoga's enduring popularity. Image Credit: Anthony Wallace Presenter: Roland Pease Producer: Harrison Lewis Assistant Producer: Sophie Ormiston

Der chinesische Forscher He Jiankui schockierte die Welt mit genmanipulierten Babys. Zunächst wurde er in China gefeiert. Doch als seine Genforschung international massiv kritisiert wurde, liess China ihn fallen. Nun forscht He Jiankui überraschend wieder. Heutiger Gast: Matthias Sander Weitere Informationen zum Thema: https://www.nzz.ch/technologie/he-jiankui-erschuf-drei-genmanipulierte-babys-jetzt-bricht-er-sein-schweigen-ld.1722020 Hörerinnen und Hörer von «NZZ Akzent» lesen die NZZ online oder in gedruckter Form drei Monate lang zum Preis von einem Monat. Zum Angebot: nzz.ch/akzentabo

In the decade since the genome editing capabilities of CRISPR-Cas9 emerged, research into novel medicines has boomed – but alongside progress comes new ethical considerations. Controversy erupted in 2018, when Chinese scientist He Jiankui created the first babies with edited genomes. After leaving prison last year, he's now back in the lab trying to raise support for new research but refuses to discuss the ethical implications of his work. Dr Joy Zhang recently arranged a bioethics seminar and invited He Jiankui, it was the first time he'd agreed to engage with a global cohort of CRISPR scientists since returning to his research. Going back in time from cutting-edge to ancient technology, some of the oldest stone tools ever used by human ancestors have been unearthed at a fossil site in Kenya. Professor Tom Plummer talks us through the findings and how important the tools were in our evolution. And we immerse ourselves in the mysterious sounds of the Arctic and Antarctic, from singing ice to the man-made noises of oil and gas drilling. These dramatic soundscapes, created for the Polar Soundscapes project, showcase just how busy our oceans are. Dr Geraint Rhys Whittaker, composer and project lead, believes a novel approach may be required to prompt climate action. Image Credit: Anthony Wallace Presenter: Roland Pease Producer: Harrison Lewis Assistant Producer: Sophie Ormiston

00:47 A metadevice for faster electronicsIn the past, increasing the speeds of electronics required designing smaller components, but further reductions in size are being hampered by increasing resistance. To get around this, researchers have demonstrated a ‘metadevice', which prevents resistance building up by concentrating the flow of signals into specific regions of the device. The hope is that this meta-method could be used to create even smaller electrical components in the future.Research article: Nikoo & Matioli06:27 Research HighlightsHow waiting times for services are higher for people in the US with low incomes, and how your brain hears an alarm while you're asleep.Research Highlight: Who wastes more time waiting? Income plays a partResearch Highlight: Noise shatters deep sleep thanks to dedicated brain circuit08:52 The research gaps in social media's impact on teen mental healthIn the last ten years, levels of social media use and reported levels of mental health issues among adolescents have both increased. There is much concern that these trends are linked, but hard evidence has been hard to come by. So how can scientists get a better understanding of what's going on? In a Comment article for Nature, researchers argue that, rather than lumping ‘young people' into one homogeneous group, future studies should consider where they are in terms of their development, as this could influence the potential impacts of social media use.Comment: How social media affects teen mental health: a missing link19:52 Briefing ChatWe discuss some highlights from the Nature Briefing. This time, we discuss self-burying devices that can plant seeds in remote areas from the air, and scientists' reactions to a talk by CRISPR-baby researcher He Jiankui.Nature Video: This device corkscrews itself into the ground like a seedNature News: Disgraced CRISPR-baby scientist's ‘publicity stunt' frustrates researchers Hosted on Acast. See acast.com/privacy for more information.

In November 2018 Chinese scientist Dr He Jiankui made a big announcement; he had illegally “edited” the DNA of Chinese twin girls Lulu and Nana in an attempt to prevent them from contracting HIV. The news made global headlines and the scientific community reacted with horror. But why is it so controversial to mess with our genes?Dr Julia Shaw is joined by geneticist Dr Adam Rutherford to discuss the dangers of gene editing and how it relates to Nazism and the dark history of trying to breed “better people”, the subject of Adam's BBC Radio 4 series Bad Blood: The History of Eugenics. CREDITS Presenters: Dr Julia Shaw and Dr Adam Rutherford Producer: Laura Northedge Assistant Producer: Hannah Ward Editor: Anna Lacey Music: Matt ChandlerCommissioning Executive: Dylan Haskins Commissioning Assistant Producer: Adam Eland#BadPeople_BBCClip: The He Lab “About Lulu and Nana: Twin Girls Born Healthy After Gene Surgery As Single-Cell Embryos”

He Jiankui has plans for finding cures for devastating genetic diseases. Should the scientific community trust him? Thanks for listening to WIRED. Check back in tomorrow to hear more stories from WIRED.com.

He Jiankui has plans for finding cures for devastating genetic diseases. Should the scientific community trust him? Thanks for listening to WIRED. Check back in tomorrow to hear more stories from WIRED.com.

A young couple have just been approved for pregnancy. They want the best future for their child. A designer lab promises that it will genetically engineer for them a basketball GOAT. Genetic engineering, basketball, designer babies, CRISPR-Cas9, He Jiankui. Support the showLearn more about Paradise Future at https://www.paradisefuturepodcast.com/Support the show at https://www.patreon.com/abeldoFollow Paradise Future on Instagram https://www.instagram.com/paradisefuturepodcast/Get in touch with Jesse Nguyenhttps://www.instagram.com/jdknewin

My special guest is Eben Kirksey, who's here to discuss his book about the fast-growing worldwide interest in manipulating human DNA. Get his book The Mutant Project: Inside the Global Race to Genetically Modify Humans on Amazon.About the book:An anthropologist visits the frontiers of the next scientific revolution to ask: Whose values are guiding gene editing experiments, and what are the implications for humanity? At a conference in Hong Kong in November 2018, Dr. He Jiankui announced that he had created the first genetically modified babies - twin girls named Lulu and Nana - sending shockwaves around the world. A year later, a Chinese court sentenced Dr. He to three years in prison for "illegal medical practice". As scientists elsewhere start to catch up with China's vast genetic research program, gene editing is fueling an innovation economy that threatens to widen racial and economic inequality. Fundamental questions about science, health, and social justice are at stake: Who gets access to gene editing technologies? As countries around the globe, from the US to Indonesia, loosen regulations, can we shape research agendas to promote an ethical and fair society? Eben Kirksey takes us on a groundbreaking journey to meet the key scientists, lobbyists, and entrepreneurs who are bringing cutting-edge genetic modification tools like CRISPR to your local clinic. He also ventures beyond the scientific echo chamber, talking to disabled scholars, doctors, hackers, chronically ill patients, and activists who have alternative visions of a future shaped by genetic engineering. The Mutant Project empowers us to ask the right questions, uncover the truth, and better prepare for this brave new world we're already entering.Follow us on InstagramFollow us on Facebook It's super easy to access our archives! Here's how: iPhone Users:Access Mysterious Radio from Apple Podcasts and become a subscriber there, or if you want access to even more exclusive content, join us on Patreon. Android Users:Enjoy over 800 exclusive member-only posts to include ad-free episodes, case files, and more when you join us on Patreon.  Please copy and Paste our link in a text message to all your family members and friends! We'll love you forever! (Check out Mysterious Radio!)

En este programa de «El mundo que se avecina», Albert Cortina y D. Miquel-Àngel Serra Beltrán, doctor en Biología e investigador en Biomedicina, continúan su conversación en torno a la «Preservación de la naturaleza humana». En esta ocasión, se centran en el mejoramiento humano que se podría realizar a través de la genética. Es cierto que nuestra tecnología va avanzando de día en día y que la preocupación del ser humano es ir mejorando su entorno. Hay muchas cosas buenas que se podrían hacer con la biotecnología, pero hay una línea roja que no se puede cruzar: las células de la línea germinal, aquellas que dan origen los óvulos y espermatozoides que posteriormente podrían convertirse en un embrión. No podemos jugar con ellas, porque afectan no solo al individuo, sino a la especie. Para ilustrar este postulado, se hace eco de la investigación de He Jiankui, científico chino que se saltó todas las leyes de la bioética para editar una gemelas chinas en la línea germinal con la técnica CRISPR/Cas9.

Directed by Cody Sheehy and produced by Samira Kiani, MAKE PEOPLE BETTER tells the story of Dr. He Jiankui, a Chinese geneticist who made waves by producing the world's first genetically-edited babies in 2018. Although Dr. He's work was feted at first by the Chinese government, the ensuing pandemonium over the ethical implications of his work quickly led to his disappearance. As the scientific community grapples with his boundary-breaking work, so too must they ask themselves whether or not there is a cost to their desire to make people better. In this 1on1, we speak to Sheehy and Kiani about the boundaries between science and ethics and changing public opinion.

The Chinese scientist who claimed to have created the world's first gene-edited babies was quietly released from prison early this month after serving a three-year sentence, state media confirmed. His release sparked a new round of discussions about the ethics of gene-editing human embryos and the unexpected harm that may occur. Lead researcher He Jiankui […]

Drei ukrainische Wissenschaftlerinnen erzählen ihre Fluchtgeschichten und wie sie an Hochschulen in der Schweiz nun Fuss fassen. Ausserdem: Auf der Suche nach der Wunderbatterie, und: Kraken sind Glucken, warum sie am Meeresgrund teils jahrelang brüten. (00:39) Meldungen: Neuvermessung des W-Boson schürt Zweifel am Standardmodell der Teilchenphysik. He Jiankui, der die ersten genmanipulierten Babies geschaffen haben will, kommt in China nach drei Jahren Haft aus dem Gefängnis. 50'000.Spinnenart entdeckt; nochmals so viele dürfte es geben. (06:43) Suche nach der Wunderbatterie: Die Batteriebranche ist im Aufwind. Und die Forschung tüftelt an immer besseren Batterien - möglichst leistungsfähig, langlebig aber auch ökologisch sollen sie sein. Gesucht ist eine eigentliche Wunderbatterie. Diese Woche nun hat eine Firma angekündigt, sie wolle im Kanton Thurgau eine riesige Fabrik bauen, in der dereinst bis zu 1000 Mitarbeiter Batteriezellen herstellen sollen - 48 Millionen Stück pro Jahr. Was ist von dieser Ankündigung zu halten und wie weit ist die Suche nach der Wunderbatterie? (12:11) Kraken-Garten in der Tiefsee: Kraken sind Glucken. Scheinbar. Manche Tiefseekraken zumindest brüten mitunter jahrelang über ihren Eiern. Nun ist klar, was ihre Brut in die Länge zieht: nicht übertriebene Mutterliebe, sondern tiefe Wassertemperaturen. Doch die cleveren Tiere haben ihre Strategien, um ungesund langes Brüten am kalten Tiefseegrund abzukürzen. (17:33) In die Schweiz geflüchtet: Forscherinnen und Studenten, die aus der Ukraine geflüchtet sind, werden in der Schweiz unterstützt. Die ersten unter ihnen sind daran, an hiesigen Hochschulen und Forschungseinrichtungen Fuss zu fassen. Wir erzählen die Geschichten von Oksana S., Svitlana Drozdovska und Oksana Ruchynksa.

Deze maand live vanuit de Arenbergschouwburg in Antwerpen! Harige zwarte gaten! De uitvinder van de GIF! De dolk van Toetanchamon! De Kawasaki Bex! Combiovens met identiteitscrisis! En nog veel meer... Shownotes: https://maandoverzicht.nerdland.be/nerdland-maandoverzicht-april-2022/ Gepresenteerd door Lieven Scheire, met Jeroen Baert, Marian Verhelst, Els Aerts, Stephanie Dehennin, Hetty Helsmoortel, Kurt Beheydt, Peter Berx, Bart Van Peer en Natha Kerkhofs. (00:00:00) Intro (00:03:14) Impact van de oorlog op het wetenschapsnieuws (00:07:20) Oneweb-lancering en de vlaggetjes op de raket (00:08:31) Ook ExoMars missie loopt mogelijk vertraging op (00:10:50) Gebruik van deepfakes in de oorlog (00:13:04) Textiel dat naar je luistert (00:19:16) RIP Stephen Wilhite uitvinder van de GIF (00:22:23) De dinosaurussen stierven in de lente (00:27:43) Smart combiovens denken opeens dat ze stoomovens zijn (00:32:52) Zwarte gaten blijken haar te hebben (00:49:54) Elon Musknieuws (00:54:50) Lieven goes Amerika (00:57:27) Musk wil vechten met Putin (01:01:05) De Kawasaki Bex, een nieuwe robot (01:06:37) Spot robot van Boston Dynamics patrouilleert in Pompei (01:08:58) Mieren bevriezen om te kijken hoe ze hun weg terugvinden (01:15:54) De dolk van Toetanchamon blijkt gemaakt van meteoriet (01:20:57) Jeroen's Niet Als Lichtzinnig Opgevatte Korte Wetenschapsnieuws (01:23:15) NERDLANDFESTIVAL komt dichterbij (01:25:47) JWST trekt mooie foto (01:26:30) He Jiankui zou binnenkort vrijkomen (01:28:11) SPONSOR Eeve en de Willow robot

#adn #genes #superhumano He Jiankui es un investigador biomédico y genético chino que tomó la decisión, engañando a las autoridades y a las parejas implicadas en el experimento, para crear los primeros embriones con humanos modificados genéticamente. Los humanos podemos tener mutaciones o variaciones genéticas, o intentar curar enfermedades de base genética heredadas o acaecidas, pero nunca antes nadie se había atrevido a alterar los genes de embriones sanos, en este caso con el "propósito humanitario", según el propio autor, de hacer nacer niños inmunes al virus del VIH que provoca la enfermedad del SIDA. De su trabajo nacieron 3 niñas, cuyo paradero y estado de salud se desconoce, que actualmente están bajo protección de China. Aunque esta es una práctica está prohibida en buena parte de occidente y también en China, el experimento de He Jiankui salió adelante y su atrevimiento le ha acostado una inhabilitación de por vida, una cuantiosa multa y que dé con sus huesos en la cárcel. Sin embargo, existen otros países, y también científicos, entre ellos Rusia, que no tienen prohibida esta práctica. La comunidad científica coincide en que es altamente posible que en el futuro nuevos investigadores lo intenten, según la tecnología avanza, por lo que las preguntas éticas al respecto se disparan: Cuando todo se perfeccione, cuando la tecnología avance, sería teóricamente posible crear humanos con capacidades superiores a la herencia genética que hasta la fecha ha presidido la evolución humana. ¿A qué nos enfrentamos? ¿Estamos ante una nueva raza superhumana? Mis Aparatos y equipos imprescindibles para trabajar y vivir: https://www.amazon.es/shop/juanfranciscocalero-clubonmotor Apóyame para hacer más y mejores vídeos en PATREON. Sé mi mentor: https://www.patreon.com/jfcalero Sígueme en INSTAGRAM, TWITTER o AMBAS, súperfácil: @jfcalero No te vayas a la cama sin saber algo más, o al menos sin saber algo nuevo. Si lo hiciste, misión cumplida, gracias por acompañarme. Puedes ver el video correspondiente a este podcast en el siguiente enlace: https://www.youtube.com/watch?v=dOn1nUICc44&ab_channel=JFCALERO-Enuncascar%C3%B3ndenuez

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Kevin Davies is a renowned British science journalist and the executive editor of The CRISPR Journal, based in New York. His literary career began with Breakthrough: The Race to Find the Breast Cancer Gene in the early 1990s, followed by Cracking the Genome, which details the dramatic story of one of the greatest scientific feats ever accomplished: the mapping of the human genome. His other titles include the $1,000 Genome, DNA: The Story of the Genetic Revolution, and his most recent release, Editing Humanity: The CRISPR Revolution and the New Era of Genome Editing, for which he won a Guggenheim Fellowship for science writing in 2017. Kevin studied at Oxford University and moved to the US in 1987 after earning his Ph.D. in genetics. He is the founding editor of the Nature Genetics journal and Bio-IT World magazine, former editor-in-chief of Cell Press, and the first publisher of C&EN, the weekly magazine of the American Chemical Society. In today's episode, Kevin elaborates on his career trajectory and explains why he believes that hanging up his lab coat was the best decision he ever made. We also touch on the common themes that run through his books, some of the challenges scientific publishers and editors face, and the importance of promoting the work of women scientists. We also cover vectors, CRISPR babies, the cost of gene therapy, and so much more! Make sure not to miss this fascinating discussion with the remarkable Kevin Davies. “How we turn this stunning 21st-century medicine into therapies that are affordable is going to be a Nobel Prize-winning discovery if anybody can crack that one.” — @KevinADavies Key Points From This Episode: Kevin's career trajectory and his so-called “desperate” shift to science journalism. How Kevin believes the field of genetics has evolved since he was a geneticist in the 1980s. Learn about the impetus behind the Nature Genetics journal and The CRISPR Journal. What motivated Kevin to write Breakthrough, including a meeting with Mary-Claire King. Three elements in all of his books: genetics, medical or societal impact, and personal drama. Hanging up his lab coat to join Nature and the access to authors that it afforded him. Kevin reflects on the demographic representation and “race to the finish line” issues in scientific publishing and the burden editors face. The lens through which Nobel Prizes are considered and how it can shift perspectives. The importance of promoting women in science, who have traditionally been overlooked. How Kevin's book, Editing Humanity, coincided with Doudna and Charpentier making history as the first two women to share a Nobel Prize. Stanley Qi's role in the CRISPR story, which Kevin calls an “unsung contribution.” Speculation and trepidation surrounding vectors: Kevin shares some new thinking. Germline genome editing, CRISPR babies, He Jiankui, and controversy in Hong Kong. Learn more about the exponential cost of gene therapies and gene editing drugs.

The CRISPR Children is a podcast series about the children whose genomes were edited before their birth in 2018. The podcasts accompany a story I did about these children in Nature Biotechnology by the same name. You can find the story here: https://rdcu.be/cB7Nx  The children were born somewhere in China. They came about due to experiments performed in the lab of He Jiankui at Southern University of Science and Technology in Shenzhen. These were unethical experiments. How are the children? And how could you assess their health and possible future risks? And why are they genetically mosaic? There is a lot of secrecy and rumor about these children. One has to maintain their privacy and dignity. They are celebrities and victims. They and their parents might be helped if the biomedical community tried to understand more about the experiments. But that is far from straightforward. Especially because many scientists declined to talk about them. But a number of them kindly did speak with me and I am grateful for that. Here is some of what I heard.       

The CRISPR Children is a series of podcasts about the children whose genomes were edited before their birth in 2018. The podcasts accompany a story I did about these children in Nature Biotechnology by the same name. You can find the story here: https://rdcu.be/cB7Nx  The children were born somewhere in China and the result of experiments performed in the lab of He Jiankui at Southern University of Science and Technology in Shenzhen. These were unethical experiments. But how are the children? And how could you assess their health and possible future risks? There is a lot of secrecy and rumor about these children. One has to maintain their privacy and dignity, of course. But they are also victims. They and their parents might be helped if the biomedical community tried to understand more about the experiments. But that is far from straightforward. Especially because many scientists declined to talk about them. But a number of them kindly did speak with me and I am grateful for that. Here is some of what I heard.  This episode is with Dr Kiran Musunuru of the University of Pennsylvania, a physician-scientist who works in genetics and gene-editing. He has also co-founded a company called Verve Therapeutics. He has written a book about the children called: The CRISPR generation The Story of the World's First Gene-Edited Babies.

Tras la investigación al genetista chino He Jiankui, por modificar los genes de dos bebés, el Gobierno indicó que será condenado por violar las leyes, falsificar permisos y continuar con su investigación para lograr el nacimiento de un tercer bebé bajo el mismo procedimiento. El doctor Elmer Huerta explica los detalles de este proceso.

Tras la investigación al genetista chino He Jiankui, por modificar los genes de dos bebés, el Gobierno indicó que será condenado por violar las leyes, falsificar permisos y continuar con su investigación para lograr el nacimiento de un tercer bebé bajo el mismo procedimiento. El doctor Elmer Huerta explica los detalles de este proceso.

What does the birth of babies whose embryos have gone through genome editing mean—for science and for all of us? In November 2018, the world was shocked to learn that two babies had been born in China with DNA edited while they were embryos—as dramatic a development in genetics as the 1996 cloning of Dolly the sheep. In this book, Hank Greely, a leading authority on law and genetics, tells the fascinating story of this human experiment and its consequences in CRISPR People: The Science and Ethics of Editing Humans (The MIT Press, 2021). Greely explains what Chinese scientist He Jiankui did, how he did it, and how the public and other scientists learned about and reacted to this unprecedented genetic intervention. The two babies, nonidentical twin girls, were the first “CRISPR'd” people ever born (CRISPR, Clustered Regularly Interspaced Short Palindromic Repeats, is a powerful gene-editing method). Greely not only describes He's experiment and its public rollout (aided by a public relations adviser) but also considers, in a balanced and thoughtful way, the lessons to be drawn both from these CRISPR'd babies and, more broadly, from this kind of human DNA editing—“germline editing” that can be passed on from one generation to the next. Greely doesn't mince words, describing He's experiment as grossly reckless, irresponsible, immoral, and illegal. Although he sees no inherent or unmanageable barriers to human germline editing, he also sees very few good uses for it—other, less risky, technologies can achieve the same benefits. We should consider the implications carefully before we proceed. Galina Limorenko is a doctoral candidate in Neuroscience with a focus on biochemistry and molecular biology of neurodegenerative diseases at EPFL in Switzerland. To discuss and propose the book for an interview you can reach her at galina.limorenko@epfl.ch. Learn more about your ad choices. Visit megaphone.fm/adchoices

In The Mutant Project: Inside the Global Race to Genetically Modify Humans (St. Martin's Press, 2020), anthropologist Eben Kirksey visits the frontiers of genetics, medicine, and technology to ask: Whose values are guiding gene editing experiments? And what does this new era of scientific inquiry mean for the future of the human species? At a conference in Hong Kong in November 2018, Dr. He Jiankui announced that he had created the first genetically modified babies—twin girls named Lulu and Nana—sending shockwaves around the world. A year later, a Chinese court sentenced Dr. He to three years in prison for “illegal medical practice.” As scientists elsewhere start to catch up with China's vast genetic research program, gene editing is fueling an innovation economy that threatens to widen racial and economic inequality. Fundamental questions about science, health, and social justice are at stake: Who gets access to gene editing technologies? As countries loosen regulations around the globe, from the U.S. to Indonesia, can we shape research agendas to promote an ethical and fair society? Eben Kirksey takes us on a groundbreaking journey to meet the key scientists, lobbyists, and entrepreneurs who are bringing cutting-edge genetic engineering tools like CRISPR to your local clinic. He also ventures beyond the scientific echo chamber, talking to disabled scholars, doctors, hackers, chronically-ill patients, and activists who have alternative visions of a genetically modified future for humanity. The Mutant Project empowers us to ask the right questions, uncover the truth, and navigate this brave new world. Galina Limorenko is a doctoral candidate in Neuroscience with a focus on biochemistry and molecular biology of neurodegenerative diseases at EPFL in Switzerland. To discuss and propose the book for an interview you can reach her at galina.limorenko@epfl.ch. Learn more about your ad choices. Visit megaphone.fm/adchoices

In The Mutant Project: Inside the Global Race to Genetically Modify Humans (St. Martin's Press, 2020), anthropologist Eben Kirksey visits the frontiers of genetics, medicine, and technology to ask: Whose values are guiding gene editing experiments? And what does this new era of scientific inquiry mean for the future of the human species? At a conference in Hong Kong in November 2018, Dr. He Jiankui announced that he had created the first genetically modified babies—twin girls named Lulu and Nana—sending shockwaves around the world. A year later, a Chinese court sentenced Dr. He to three years in prison for “illegal medical practice.” As scientists elsewhere start to catch up with China's vast genetic research program, gene editing is fueling an innovation economy that threatens to widen racial and economic inequality. Fundamental questions about science, health, and social justice are at stake: Who gets access to gene editing technologies? As countries loosen regulations around the globe, from the U.S. to Indonesia, can we shape research agendas to promote an ethical and fair society? Eben Kirksey takes us on a groundbreaking journey to meet the key scientists, lobbyists, and entrepreneurs who are bringing cutting-edge genetic engineering tools like CRISPR to your local clinic. He also ventures beyond the scientific echo chamber, talking to disabled scholars, doctors, hackers, chronically-ill patients, and activists who have alternative visions of a genetically modified future for humanity. The Mutant Project empowers us to ask the right questions, uncover the truth, and navigate this brave new world. Galina Limorenko is a doctoral candidate in Neuroscience with a focus on biochemistry and molecular biology of neurodegenerative diseases at EPFL in Switzerland. To discuss and propose the book for an interview you can reach her at galina.limorenko@epfl.ch. Learn more about your ad choices. Visit megaphone.fm/adchoices

As I said in a BreakPoint commentary last month, gene-editing technologies such as CRISPR and what's being called “Prime Editing” are “existential threats.” We have no idea what our attempts to play god with the human genome will unleash on humanity. Yet, we insist on charging ahead despite our imperfect knowledge with an unbounded confidence in our abilities. Coming from a concerned non-scientist like me, these concerns can be easily dismissed as alarmist, but what if the concern comes from the Director of the National Institutes of Health? It turns out that Francis Collins is also concerned. In a recent article in Discover Magazine entitled “We Must Never Allow Our Technology to Eclipse Our Humanity,” Collins called for a “moratorium of at least five years on heritable human gene editing.” Heritable gene editing technologies, like “Prime Editing,” aim to edit genes that can be passed on to future generations, along with any unintended and dangerous mutations. This differs from “non-heritable gene editing,” which can be used to treat people with “life-threatening disorders, such as sickle cell disease, HIV infection, cancer and muscular dystrophy.” Proponents of Prime Editing talk about the possibility of making “any kind of DNA change that anyone wants at just about any site in the human genome.” Thus, according to Collins, “scientists and leaders around the globe have an obligation to consider the appropriate use — if any — of heritable human gene editing. This involves scrutinizing the safety of such experiments, including the risk of unintended mutations, as well as a clear-eyed analysis of actual medical need.” Anticipating some objections, the NIH Director added that “the current arguments — that the benefits outweigh the risks — are surprisingly uncompelling.” Finally, Collins insisted that “We must weigh the profound social, ethical and moral issues associated with modifying the germline in ways that could change the human species forever.” It's good to hear someone as prominent as the director of the National Institutes of Health voice many of the same concerns we have at BreakPoint. But, as Wesley J. Smith pointed out at National Review, it's probably not enough. As Smith notes, when it comes to “the rapid development of the most powerful technologies ever invented — CRISPR germline gene editing, “artificial life,” “3-parent” embryos, cloning,” the Trump Administration has been, and I am quoting Smith here, “derelict.” For the most part, while NIH Director Collins' statement should be applauded, it is an exception. As Smith states, “unless leaders higher up the food chain engage the question in more amplified media venues than Discover,” Collins' proposed moratorium will never happen. In some ways, as I recently pointed out, the Communist Party of China, by sentencing Dr. He Jiankui to three years imprisonment for experimenting on fetuses using CRISPR, has demonstrated more commitment to reining in scientific hubris than our own government. Of course, given their track record, it would be silly to think that Beijing cares at all about humanity dignity and the sanctity of human life. Their reaction was almost certainly because Dr. He's transgressions portrayed that country in an especially bad light. In our country, scientists differ from Dr. He in only one respect: They're a lot more subtle about what they are doing than Dr. He was. We need much more evidence that those “higher up the food chain” in this administration care about the issue and are willing to make it a priority. For that to happen, we have let them know that it's a priority for us. Gene editing, what Smith has called “biotech anarchy,” is among the greatest threats to the sanctity and dignity of human life that we currently face. I will say it again: It is an existential threat. It's time for Christians, and through them our leaders, to treat it like one.  

aclaramos el polémico caso de CRISPR con Lluís Montoliu El investigador He Jiankui y dos de sus colaboradores han sido condenados a penas de cárcel, multas económicas e inhabilitación profesional por haber editado genéticamente tres bebés en Shenzhen (China). He Jiankui ha sido condenado a tres años de cárcel, al pago de una multa de 3 000 000 de yuanes (unos 384 000 euros) y ha sido inhabilitado de por vida para trabajar en cualquier investigación que involucre embriones humanos, reproducción humana o cualquier otro aspecto de salud humana. Lluís Montoliu nos explica los detalles de este rocambolesco caso. Recomiendo la lectura de su excelente pieza en The Conversation. La música de este episodio es Schubert Fantasy in C major 'Wanderer', Op. 15, puedes encontrarla en musopen.org Este contenido es gratis y sólo te pido que, si te ha gustado, entretenido, iluminado de algún modo, lo compartas en tus redes y nos valores en tu plataforma de pódcast favorita. Gracias ;)

Confira as principais notícias internacionais desta terça-feira (31/12/19) no EstadãoSee omnystudio.com/listener for privacy information.

We're gettin' nerdy this week, people! We're donning our lab coats and venturing into a scientific conversation we have no business discussing: CRISPR/Cas9. Which is why we brought in the brain trust - i.e., Kishore Hari, science guru over at Tested.com and host of the Inquiring Minds podcast. We start off with a bit of context and discuss what CRISPR/Cas9 actually is. What's the basic science behind the technology and process, how does it work, what's its promise, and what are its dangers? We also touch on recent, very real-world events in which Chinese scientist He Jiankui announced that he had used the technology to genetically modify a pair of twin girls. We then talk to author Robin Cook whose newest novel, Pandemic, is a thriller about the unintended side effects caused by genetic engineering and the CRISPR technology. Throughout his career, Cook has had an uncanny ability to be incredibly timely with his novels. Pandemic is no different. We talk to Cook about how the black market in human organs (a theme in Pandemic) has changed in the 40 years since he wrote Coma, why it wasn't much of a surprise that someone used CRISPR/Cas9 to genetically modify humans, where we go from here, the continuing ignorance about vaccines, and how medicine and public health will change over the next generation.

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Corey and Steve discuss news of gene edited babies in China, and the future of human genetic engineering.▶️ Watch: CRISPR Babies — Episode #1Resources Generation CRISPR? He did it: He Jiankui talk at HKU conference on gene editing Economist Radio podcast interview on Genomic Prediction Transcript

On the show today we welcome Professor Henry Greely from Stanford to talk a about the recent developments in the genetic field and particularly the latest controversy around CRISPR technology. Professor Greely is the Director at the Center for Law and Biosciences at Stanford, he's a Professor of Genetics at the Stanford School of Medicine, and a Chair at the Steering Committee of the Center for Biomedical Ethics. Inside this episode we get a background on gene-editing and CRISPR technology. Prof. Greely breaks down the basic story of Dr. He Jiankui, a Chinese scientist who conducted a gene-editing experiment with twin babies, causing serious concerns in the scientific community and raising ethical questions. From there we discuss alternatives to CRISPR and ways in which the technology could be utilized more conscientiously in the future. In this context, we will also give some attention to the global position of China and the political landscape. For all this and more be sure to tune in!

El genetista chino He Jiankui trajo al mundo las gemelas Lulu y Nana cuyos embriones fueron modificados para ser inmunes al VIH. Durante una conferencia en Hong Kong, el científico explicó el alcance de su investigación que ha generado gran controversia en el mundo de la medicina. El doctor Elmer Huerta explica los detalles del estudio.

El genetista chino He Jiankui trajo al mundo las gemelas Lulu y Nana cuyos embriones fueron modificados para ser inmunes al VIH. Durante una conferencia en Hong Kong, el científico explicó el alcance de su investigación que ha generado gran controversia en el mundo de la medicina. El doctor Elmer Huerta explica los detalles del estudio.