Podcast appearances and mentions of Gene editing

For the first time in human history, we can diagnose thousands of genetic diseases—often for under $1,000—but we still can't treat most of them. The problem isn't understanding what's broken; it's delivering the fix to the right cells.Eric Kelsic, CEO of Dyno Therapeutics, joins a16z's Jorge Conde to explain how AI-designed protein shells are solving gene therapy's delivery crisis. They explore why Huntington's patients can now get 15 extra years of healthy life, how Dyno inverted the liver-to-brain delivery ratio by 1000x, and why capsids evolved by nature are now being designed by machine learning models trained on millions of variants.Eric introduces the concept of genetic agency—humanity's first-ever ability to take action at the DNA level—and details why solving delivery for common diseases will make ultra-rare disease treatments economically viable. Plus: what happens when gene therapy requires neurosurgery today but could be a simple injection tomorrow, why recent deaths in clinical trials prove we need better technology now, and how genetic medicine could become as routine as surgery within our lifetimes. Resources:Follow Eric on X: https://x.com/ekelsicFollow Jorge on X: https://x.com/JorgeCondeBioLearn more about GATC 2025: https://www.dynotx.com/gatc2025 Stay Updated:If you enjoyed this episode, be sure to like, subscribe, and share with your friends!Find a16z on X: https://x.com/a16zFind a16z on LinkedIn: https://www.linkedin.com/company/a16zListen to the a16z Podcast on Spotify: https://open.spotify.com/show/5bC65RDvs3oxnLyqqvkUYXListen to the a16z Podcast on Apple Podcasts: https://podcasts.apple.com/us/podcast/a16z-podcast/id842818711Follow our host: https://x.com/eriktorenbergPlease note that the content here is for informational purposes only; should NOT be taken as legal, business, tax, or investment advice or be used to evaluate any investment or security; and is not directed at any investors or potential investors in any a16z fund. a16z and its affiliates may maintain investments in the companies discussed. For more details please see a16z.com/disclosures.  Hosted by Simplecast, an AdsWizz company. See pcm.adswizz.com for information about our collection and use of personal data for advertising.

Could studying the DNA of extinct animals – or even bringing them back to life – help us save today's endangered species and inform modern medicine?  That may sound like the premise for a Hollywood movie, but it's work that our Raise the Line guest, Dr. Beth Shapiro, is actually engaged in as Chief Science Officer at Colossal Biosciences, which describes itself as the world's first and only de-extinction company.  “It's not just about learning about the past. It's learning about the past so we have more validated scientific information that we can use to predict what we can do to better influence the future,” she tells host Michael Carrese. An internationally-renowned evolutionary molecular biologist and paleogeneticist, Dr. Shapiro is a pioneer in ancient DNA research and has successfully sequenced genomes, like that of the dodo, to study evolution and the impact on humans. At Colossal Biosciences, she leads teams working to bring back traits of extinct species such as the mammoth, not for spectacle, but to restore ecological balance. “When species become extinct, you lose really fundamental interactions between species that existed in that ecosystem. By taking a species that's alive today and editing its DNA so that it resembles those extinct species, we can functionally replace those missing ecological interactions.” Tune into this utterly fascinating conversation to hear about what Jurassic Park got wrong, the positive ecological impact of reintroducing giant tortoises to Mauritius, and the ethics of using gene editing and other biotechnologies. Mentioned in this episode:Colossal Biosciences If you like this podcast, please share it on your social channels. You can also subscribe to the series and check out all of our episodes at www.osmosis.org/podcast

Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we explore a series of groundbreaking advancements and strategic collaborations that promise to transform drug development and patient care.In the autoimmune space, Boehringer Ingelheim has made a significant move by securing a $570 million agreement with CDR-Life. This deal centers on a trispecific antibody, a novel therapeutic approach that targets multiple pathways simultaneously, potentially revolutionizing treatments for autoimmune diseases. Boehringer's commitment to these cutting-edge modalities highlights their strategy to leverage novel technologies for more effective therapeutic solutions.Similarly, Celltrion has entered a $744 million collaboration with Kaigene, focusing on two preclinical autoimmune drugs. This partnership marks Celltrion's strategic shift from biosimilars to novel biologics, positioning the company at the forefront of biologic therapeutics. By investing in early-stage research, Celltrion aims to introduce transformative therapies for autoimmune conditions, showcasing the industry's willingness to bet on groundbreaking scientific advancements.In gene editing, Azalea Therapeutics is gaining attention with its focus on permanent genome editing using a dual-vector approach. Backed by $82 million in funding and support from CRISPR pioneer Jennifer Doudna, Azalea is poised to develop potentially curative solutions through single-dose treatments. The credibility lent by a Nobel laureate adds anticipation to their research outcomes, with the potential to significantly impact gene therapy.Shifting focus to clinical trials, Sarepta Therapeutics faces challenges after missing the primary endpoint in its confirmatory trial for Duchenne muscular dystrophy drugs. Despite this setback, Sarepta is pursuing full FDA approval, emphasizing the complex interplay between clinical data and regulatory strategies. This situation underscores the critical importance of robust confirmatory trials in securing drug approvals and ensuring patient access to new therapies.Merck is making strategic moves in oncology by regaining full control over an early-phase asset and securing $700 million from Blackstone for its oncology pipeline. This dual focus on asset acquisition and financial fortification reflects Merck's aggressive growth strategy aimed at expanding its cancer treatment offerings.Emerging from stealth mode, Neok Bio has secured a $75 million investment to advance bispecific antibody-drug conjugates into clinical trials. These bispecific ADCs represent the forefront of targeted cancer therapies, aiming for precision targeting of cancer cells while minimizing off-target effects. Neok Bio's progress could significantly enhance oncology treatment paradigms through improved therapeutic indices.Turning to regulatory landscapes, Teva's recall of over half-a-million bottles of prazosin hydrochloride due to potential carcinogenic impurities highlights ongoing challenges in ensuring drug safety and quality control within manufacturing processes. Such recalls underscore the critical importance of maintaining high standards in pharmaceutical production.In broader industry developments, we see dynamic trends where scientific innovation meets strategic business decisions and regulatory considerations. The potential impact on patient care is profound, with breakthroughs in autoimmune treatments, gene editing technologies, and targeted cancer therapies poised to alter therapeutic landscapes significantly.UCB has achieved another milestone with FDA approval for Kygevvi, an ultra-rare disease medication marking their third approval in rare conditions within three years. This success underscores UCB's strategic focus on niche markets that offer less competition but significant patient impact. Advancements in genetic research aSupport the show

According to the Bloomberg School of Public Health at Johns Hopkins University, women make up 70% of the global healthcare workforce but hold only about 25% of leadership positions. Our guest today on Raise the Line, Dr. Roopa Dhatt, has been a leading voice in the movement to correct that imbalance through co-founding an organization called Women in Global Health (WGH), which has established chapters in over 60 countries since it started a decade ago. Dr. Dhatt is also pursuing that agenda and addressing other pressing issues in healthcare as a Young Global Leader at the World Economic Forum. “We're changing the equation so women delivering health are also viewed and valued as leaders,” says the internal medicine physician and assistant professor at Georgetown University School of Medicine. Beyond leadership equity, Dr. Dhatt is also seeking to address systemic pay inequities and high levels of violence and harassment experienced by women in the health sector, issues that were highlighted in research conducted by WGH. Although WGH has seen high-level success influencing policy at the World Health Organization and United Nations, Dr. Dhatt says the heart of its success is local. “Women community health workers have begun to see themselves as leaders and the heroines of health in their communities. That's profound change.” Join host Michael Carrese for a probing conversation that identifies the structural barriers blocking advancement for women and that explains why the health of communities and the planet depend on inclusive leadership.Mentioned in this episode:Women in Global HealthWHO Report: Delivered By Women, Led By MenDr. Roopa Dhatt on LinkedIn If you like this podcast, please share it on your social channels. You can also subscribe to the series and check out all of our episodes at www.osmosis.org/podcast

Here's a polished podcast blurb suitable for LinkedIn, BlueSky, or Apple Podcasts listings — written in the JIMD Podcast tone and style: ⸻ It's one of the most talked-about breakthroughs of 2025, a first-in-human demonstration of in vivo gene editing to treat an inherited metabolic disease. In this episode, Kiran Musunuru and Rebecca Ahrens-Nicklas are joined by Julien Baruteau to unpack what this means for the field. They explore the science behind gene editing, the importance of ethical design, and the emotional weight of stopping therapy once enzyme function is restored. The conversation bridges the NEJM landmark paper (Musunuru et al., 2025) and the accompanying JIMD editorial (Rahman & Baruteau, 2025), reflecting on what this moment tells us about the future of metabolic medicine and how ready we are for it. First in Human Gene Editing for an Inherited Metabolic Disease Shamima Rahman, Julien Baruteau https://doi.org/10.1002/jimd.70056 Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease Kiran Musunuru, et al https://www.nejm.org/doi/10.1056/NEJMoa2504747

In this week's podcast, some of Britain's biggest supermarkets are refusing to engage with a review of UK farm assurance schemes.A progress report on improving schemes like Red Tractor has struggled to gather input from Britain's big retail chains.Just three supermarkets have responded on an individual basis with the British Retail Consortium (BRC) refusing to participate.Could a breakthrough by UK scientists mean the end of classical swine fever?And we find out why the world needs more red meat.This episode of the Farmers Weekly Podcast is co-hosted by Johann Tasker, Louise Impey and Hugh Broom.Contact or follow Johann (X): @johanntaskerContact or follow Louise (X): @louisearableContact or follow (X): @sondesplacefarmFor Farmers Weekly, visit fwi.co.uk or follow @farmersweeklyTo contact the Farmers Weekly Podcast, email podcast@fwi.co.uk.In the UK, you can also text the word FARM followed by your message to 88 44 0.

“They say it takes a village to raise a child. I really think it takes a village to treat a patient,” says Dr. Lanae Mullane, a naturopathic doctor and clinical strategist who has spent years at the forefront of bridging functional medicine, nutraceutical development, and digital health. In this episode of Raise the Line, host Lindsey Smith explores Dr. Mullane's view that naturopathic medicine complements conventional care by expanding -- not replacing -- the clinical toolkit, and that collaboration should be the future of medicine. “At the end of the day, collaboration and connection create the best outcomes for the people we serve,” she says. Their in-depth conversation also spans the shifting landscape of women's hormone health, including the perimenopausal transition and long-overdue calls for research equity. “We're not just smaller versions of men. We need to have dedicated research for us.” Tune in to learn about the importance of grounding health in sustainable habits, rethinking midlife care for women, and how to help patients take ownership of their health.Mentioned in this episode:Joi + BlokesSuppCoDr. Mullane's Clinical Website If you like this podcast, please share it on your social channels. You can also subscribe to the series and check out all of our episodes at www.osmosis.org/podcast

“It's kind of a miracle, frankly,” says Dr. John Buse, a distinguished professor at the University of North Carolina School of Medicine, referring to the effectiveness of GLP-1 receptor agonist medications such as Ozempic in treating type 2 diabetes, promoting significant weight loss, and reducing cardiovascular risk. As a physician scientist for the last three decades at UNC, Dr. Buse has played a key role in ushering in this new era of diabetes care, leading or participating in over 200 clinical studies on this class of drugs and others. “Nothing has impacted diabetes care like the GLP-1 receptor agonists. I have lots of patients whose diabetes was never well controlled who have seen all their metabolic problems essentially resolved.”  In this fascinating conversation with Raise the Line host Lindsey Smith, Dr. Buse not only explains how these drugs work, but also provides a clear-eyed look at side effects, and addresses issues of cost and access. Join us for the remarkable story – including the role played by Gila monsters -- behind one of the biggest developments in medicine over the past several years from a world renowned diabetes researcher and clinician. Mentioned in this episode:UNC School of Medicine If you like this podcast, please share it on your social channels. You can also subscribe to the series and check out all of our episodes at www.osmosis.org/podcast

“It wasn't a profession, it was a way of life,” observes internationally respected psychiatrist Dr. Nasser Loza, reflecting on a century-long family legacy in mental health care that began when his grandfather founded The Behman Hospital in Cairo. In this candid Raise the Line conversation with host Michael Carrese, Dr. Loza traces the transformation of psychiatry he's witnessed in his long career as increases in classifications, payment bureaucracy, reliance on pharmaceuticals, and technological disruption have each left their mark. The cumulative costs associated with these changes have, he laments, pushed care out of reach for many and hindered the human connection that is key to the discipline. He describes his prescription for countering these trends as a focus on effective and modest aims. “Rather than saying, come and see me in therapy for five years and I will make a better person out of you, I think focusing on symptom-targeted help is going to be what is needed.”  In this wide-ranging interview, you'll also learn about progress on advancing the rights of mental health patients and lowering stigmas, how to manage the rise of online therapy and use of AI chatbots, and the importance of empathy and transparency in mental health counseling. Don't miss this valuable perspective on a critically important dimension of healthcare that's informed by decades of experience as a clinician, government official and global advocate. Mentioned in this episode:The Behman HospitalMaadi Psychology Center If you like this podcast, please share it on your social channels. You can also subscribe to the series and check out all of our episodes at www.osmosis.org/podcast

Each week on Patriot Power Hour, Ben ‘The Breaker of Banksters' and Future Dan explore the latest Liberty, Security, Economic & Natural news, providing the situational awareness needed to execute your preparedness plans. Questions, Feedback, News Tips, or want to be a Guest? Reach out!Ben “The Breaker of Banksters” @BanksterBreaker on XFuture Dan@FutureDanger6 on XGet Prepared with Our Incredible Sponsors! Survival Bags, kits, gear www.limatangosurvival.comEMP Proof Shipping Containers www.fardaycontainers.comThe Prepper's Medical Handbook Build Your Medical Cache – Welcome PBN FamilyPack Fresh USA www.packfreshusa.comSupport PBN with a Donation https://bit.ly/3SICxEq

“When I was in medical school, no one had even heard of mitochondrial disease. Today, every student who graduates here knows what it is and has seen a patient with it,” says Dr. Mary Kay Koenig, director of the Center for the Treatment of Pediatric Neurodegenerative Disease at UTHealth Houston McGovern Medical School. That remarkable change in awareness has been accompanied by advances in genetic sequencing, the development of clinical guidelines, and the emergence of potential treatments in some forms of mitochondrial disease. In fact, Dr. Koenig's multidisciplinary team at UTHealth's Mitochondrial Center of Excellence has been a key player in clinical trials that may yield the first FDA-approved treatments for it. As you'll learn in this Year of the Zebra conversation with host Michael Carrese, her work in neurodegenerative diseases also includes tuberous sclerosis, where advanced therapies have replaced the need for repeated surgeries, and Leigh Syndrome, which has seen improvements in diagnoses and supportive therapies leading to better quality of life for patients.  Tune in as Dr. Koenig reflects on an era of progress in the space, the rewards of balancing research, teaching and patient care, and the need for more clinicians to center listening, humility and honesty in their approach to caring for rare disease patients and their  families.Mentioned in this episode:Mitochondrial Center of ExcellenceCenter for the Treatment of Pediatric Neurodegenerative Disease If you like this podcast, please share it on your social channels. You can also subscribe to the series and check out all of our episodes at www.osmosis.org/podcast

On today's Good Day Health Show - ON DEMAND…Host Doug Stephan and Dr. Ken Kronhaus of Lake Cardiology (352-735-1400) cover a number of topics affecting our health. First up, Doug and Dr. Ken discuss the biggest news stories in the medical world, starting with the good news of a gene-editing technique that could correct a mutation responsible for a certain type of inherited heart disease. This is a significant step forward that could offer a potential targeted correction of genetic faults. In other heart news, and not good news, there are more heart complications in pregnant women — which could be related to higher rates of obesity, women getting pregnant at an older age, and a steady increase in heart-related health problems during pregnancy. Moving on, a discussion on the growing preference of marijuana use over alcohol, and whether or not either is OK in what kind of moderation. In the good news department, there's a new blood test for cancer detection in your head and neck, a successful pig liver transplant, and AI-powered wearable devices could be the future of medicine for Parkinson's patients to offer better treatment decisions. Moving on, research shows young students who spend excessive time on social media perform worse on tests in subjects like reading, memory and language.Then, a focus on the antimicrobial resistance with some bacterial infections becoming drug-resistant, turning routine infections into deadly instances. Website: GoodDayHealthrShow.com Social Media: @GoodDayNetworks

If you needed a life-saving transplant, would you take a gene-edited organ from a pig? It sounds like science fiction, but this procedure has been researched for decades, and it could help solve the massive organ shortage. But is it ethical to use animals for their parts? And are there other risks? 

“Giving learners options gives them a better learning experience. It's more holistic and more comprehensive,” says Sean Moloney, CEO and founder of EmbodyXR, an extended reality platform focused on the use of immersive technologies in medical education. In this eye-opening Raise the Line conversation, Moloney explains how AI-powered extended reality (XR) --which integrates augmented, virtual, and simulation-based environments -- allows learners to interact with patients, explore multiple diagnostic choices, and experience varied outcomes based on their decisions. The result, he notes, is not only stronger engagement in learning, but a measurable improvement in understanding. Despite these gains, Moloney is quick to point out that he sees these technologies as complements to traditional training, not substitutes for it. “We'll never replace in-person teaching,” he says, “but we can make learners even better.” Beyond training future clinicians, the EmbodyXR platform is also offering new modes of patient and caregiver education, such as augmented reality guidance for using medical devices at home. Join host Lindsey Smith as she explores how EmbodyXR achieves and maintains clinical accuracy, the connectivity it offers between headsets, personal computers and mobile devices, and other capabilities that are shaping the future of how healthcare professionals and patients will learn. Mentioned in this episode:EmbodyXR If you like this podcast, please share it on your social channels. You can also subscribe to the series and check out all of our episodes at www.osmosis.org/podcast

Why has America struggled so much to effectively manage the opioid use crisis? One of the answers, as you'll learn in this eye-opening episode of Raise the Line, is rooted in laws and attitudes from the early 20th century that removed addiction from the realm of medicine and defined it as a moral failing.  “The federal Harrison Act of 1914 forbade any physician from prescribing opioids to people with addiction, so it became more the purview of law enforcement or behavioral health or religion,” says Dr. Melody Glenn, who regularly confronts the consequences of this history during shifts in the emergency department at Banner-University Medical Center in Tucson, Arizona. And as Glenn explains to host Caleb Furnas, the resulting stigma associated with addiction has extended to the treatments for it as well, especially methadone, despite its effectiveness. Drawing on her dual expertise in emergency and addiction medicine, Glenn dispels misconceptions that medication-assisted treatment merely replaces one addiction with another, and emphasizes that harm reduction is critical to saving lives. Her desire to break prevailing stigmas led her to discover the story of Dr. Marie Nyswander, who pioneered methadone maintenance therapy in the 1960s and is featured in Dr. Glenn's new book, Mother of Methadone: A Doctor's Quest, a Forgotten History, and a Modern-Day Crisis. You'll leave this instructive interview understanding the roots of our flawed approach to addiction treatment, meeting an overlooked pioneer in the field, and admiring a devoted and compassionate physician who is following in her footsteps.  Mentioned in this episode:Banner-University Medical CenterMother of Methadone book If you like this podcast, please share it on your social channels. You can also subscribe to the series and check out all of our episodes at www.osmosis.org/podcast

When baby KJ was diagnosed with a rare metabolic disorder, the team at Children's Hospital of Philadelphia (CHOP) had a unique treatment option for him: a personalized gene editing therapy developed in collaboration with Penn Medicine. On this episode, Madeline speaks to CHOP geneticist Dr. Rebecca Ahrens-Nicklas, who describes how KJ's first-of-its-kind treatment was developed – and what this amazing breakthrough means for other patients. 

“We don't view a person with chronic pain as someone who has a chronic illness and the effect of that is we can't follow patients continuously over prolonged periods of time,” says Dr. Jacob Hascalovici, a neurologist and pain specialist based in New York City.  In co-founding Bliss Health, Dr. Jacob, as he is known, has set out to create a continuous care model for chronic pain treatment that matches the approach taken for patients with diabetes or high blood pressure. The Bliss Health formula includes an initial meeting with a physician that produces a care plan; remote therapeutic monitoring on an ongoing basis; and a monthly meeting with a nurse to review data and determine next steps, including additional appointments with physicians as needed.  All of this occurs via a digital platform which provides a welcome option for patients with mobility issues and can fill gaps in access to specialists, especially in rural areas. Dr. Jacob is also hoping to make chronic pain patents feel respected, which is not always the case in their encounters with the healthcare system. “Because pain is not something that can be seen or measured, oftentimes patients feel marginalized, dismissed and disempowered by providers.” Join Raise the Line host Lindsey Smith for a valuable conversation that also touches on policy changes that could strengthen telemedicine, and has details on the first non-opioid based pain medication to receive FDA approval in over 20 years.Mentioned in this episode:Bliss Health If you like this podcast, please share it on your social channels. You can also subscribe to the series and check out all of our episodes at www.osmosis.org/podcast

In recent months, public health advocates in the United States have raised concerns about proposed changes to vaccine policy, cuts to food assistance programs, rollbacks of environmental protections and reductions in public health staffing. Chief among them has been Dr. Georges Benjamin who, as executive director of the American Public Health Association (APHA) since 2002, has led national efforts to create a healthier America. Raise the Line host Lindsey Smith recently sat down with Dr. Benjamin to understand more about the current state of public health and explore the path forward, and learned that a top priority for APHA is battling the misinformation that Dr. Benjamin believes is fueling support for many of these changes. “The challenge we have right now is that as a society, we've gone into our little corners and live in our own ecosystems. More people are getting their information from a single source and they're not validating that information to make sure that it's true.” Tune into this thoughtful and timely conversation to hear Dr. Benjamin's advice for curbing the spread of misinformation, how APHA is trying to help people understand the value of public health initiatives, and what the U.S. can learn from other countries about improving public health. Mentioned in this episode:American Public Health Association If you like this podcast, please share it on your social channels. You can also subscribe to the series and check out all of our episodes at www.osmosis.org/podcast

In this episode, I talked to Dr. Alexander Marson, cofounder of Arsenal Biosciences and director of the Gladstone-UCSF Institute of Genomic Immunology. He is one of the pioneers of non-viral T-cell editing and is a serial biotech entrepreneur. We talked about Car T-cell Therapy, using CRISPR to edit T-cells, biotech, and his collaboration with Sean Parker, cofounder of Napster. ---------------------------------------------------------------Thanks to the sponsors:Audible: Use my link for a 30-day free trial: http://audibletrial.com/diamondgoatNewsly: https://newsly.mepromo code to receive a 1-month free premium subscription: EARLYMORNING Libysn: https://libsyn.compromo code: DG Dubby Energy: https://www.dubby.ggpromo code for 10% off: DIAMONDGOATOpus Clips:  https://www.opus.pro/?via=diamondgoat----------------------------------------------------------------------------------Listen on:Podcast website: https://www.spreaker.com/podcast/dg-early-morning-show--5943922Spotify: https://open.spotify.com/show/0EuhA6WyuerHtVAqcFrFeOPodcast YT channel clips: https://www.youtube.com/@dgearlymorningshowTiktok: @dgearlymorningshowApple Podcast: https://podcasts.apple.com/us/podcast/dg-early-morning-show/id1575451533Amazon Music: https://music.amazon.com/podcasts/f050b86c-1dad-4bc3-b12f-6aa5fa62438c  Goodpods: https://goodpods.com/podcasts/dg-early-morning-show-211830RadioPublic: https://radiopublic.com/dg-earlymorning-show-WoML4rBreaker: https://www.breaker.audio/dg-early-morning-showReason: https://reason.fm/podcast/dg-earlymorning-show--------------------------------------Check out my other stuff:Instagram: @itzdiamondgoatTwitter: @lildiamondgoatMain YT channel: youtube.com/diamondgoatTiktok: @lildiamondgoatSoundcloud: @Lil DiamondgoatSpotify: @Lil DiamondgoatMerch store: https://diamondgoat.creator-spring.com

“Probably the most exciting thing I've seen in gene therapy over the last ten years is we now have a lot of tools for selective delivery, which will hopefully make treatments more safe and a lot more successful,” says Dr. Jessica Duis, a geneticist and pediatrician focused on the management of individuals with complex, rare disorders. Dr. Duis, who has worked on several gene therapies that are now approved or progressing through the accelerated approval pathway, is currently VP of Clinical Development at GondolaBio, a clinical-stage biopharmaceutical company focused on developing therapeutics for genetic diseases. As you'll learn in this Year of the Zebra episode with host Lindsey Smith, Dr. Duis is encouraged by other recent advances in genetic technology as well, and thinks momentum will grow as breakthrough treatments emerge. “I think we're hopefully going to continue to see companies that are working in rare disease be more successful and really drive how regulators think about making decisions in terms of bringing treatments to patients. I think we're at the tip of the iceberg in terms of the future of truly transformational therapies.”  This wide ranging conversation also explores Dr. Duis' team approach to patient care, her work on clinical endpoints, the importance of patient communities, and her book series, Rare Siblings Stories.Mentioned in this episode:GondolaBioRareDiseaseDocElsevier Healthcare Hub on Rare DiseasesRare Sibling Stories If you like this podcast, please share it on your social channels. You can also subscribe to the series and check out all of our episodes at www.osmosis.org/podcast

Guest: Alexis Leonard With two FDA-approved gene therapies, including the first CRISPR-based treatment, curative strategies for sickle cell disease are continuing to expand the treatment landscape. However, challenges remain in optimizing safety and accessibility, particularly for patients unable to tolerate current myeloablative conditioning. Learn about advancements in ex vivo editing, such as improved stem cell targeting and multiplex editing, and the possibility of in vivo approaches that may help transform sickle cell disease management with Dr. Alexis Leonard, who works in the Department of Hematology at St. Jude's Hospital in Memphis, Tennessee.

Guest: Alexis Leonard CRISPR technology, while relatively new, has shown potential in treating patients with sickle cell disease by increasing fetal hemoglobin. Though this gene therapy comes with safety considerations, refinements and advancements in CRISPR technology can offer new management options for sickle cell disease patients. Learn about the history of CRISPR as a sickle cell disease treatment and the future of this strategy with Dr. Alexis Leonard, who works in the Department of Hematology at St. Jude's Hospital in Memphis, Tennessee.

TWiV explains experiments to create genetically modified chickens that are resistant to avian influenza infection, and discovery of combination antiviral therapy for HTLV-1 infection. Hosts: Vincent Racaniello, Alan Dove, and Rich Condit Subscribe (free): Apple Podcasts, RSS, email Become a patron of TWiV! Links for this episode Support science education at MicrobeTV Creating avian influenza virus resistant chickens (Nat Comm) Antiretroviral therapy and apoptosis inhibition for HTLV-1 (Cell) HTLV-1 infection (Cleveland Clinic) Letters read on TWiV 1257 Timestamps by Jolene Ramsey. Thanks! Weekly Picks Rich – Chagas Disease, an Endemic Disease in the United States Alan – Archive of molecular biology artifacts ends its long, strange trip at a museum Vincent – The World's Oceans Are Hurtling Toward Breaking Point Intro music is by Ronald Jenkees Send your virology questions and comments to twiv@microbe.tv Content in this podcast should not be construed as medical advice.

With nearly one in ten newborns in the US requiring care in a Neonatal Intensive Care Unit, the importance of NICUs has never been more clear. On today's episode of Raise the Line, we're shining a light on the extraordinary world of NICUs with Lindsay Howard, a veteran nurse with over 17 years of experience caring for premature and critically ill infants. She currently works in a Level IV NICU at Children's Memorial Hermann Hospital in Houston, one of the most advanced neonatal units in the country. “We call ourselves ‘the ER of the neonate world' because we're never full. We have to make space no matter what comes in off the street, and at the biggest medical center in the world, we see all the things,” she explains. In this enlightening conversation with host Lindsey Smith, Howard describes how advances in medicine have made it possible to provide more types of care for younger and smaller babies, creating a need for NICU nurses to develop subspecialties. In her case, Howard is on a dedicated team that handles the placement and maintenance of all central line IVs, and has earned certifications in neonatal and pediatric chemotherapy and biotherapies. “We see babies that we may not have seen before being born with cancerous tumors who need chemotherapy to try and eliminate it, or just give them more time with their family.” This is a revealing look inside the workings of a top tier NICU where you'll learn about approaches to care that support healthy neurodevelopment, how clinical staff handle the emotional challenges of the job, and how her own experience as a mother with twins needing NICU care impacted her work.  Mentioned in this episode:Children's Memorial Hermann Hospital If you like this podcast, please share it on your social channels. You can also subscribe to the series and check out all of our episodes at www.osmosis.org/raisethelinepodcast

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“When you think about where we were as a country before Medicare and Medicaid were created and where we are now, it's an incredible story,” says Chiquita Brooks-LaSure, who until earlier this year was the administrator for the Centers for Medicare and Medicaid Services (CMS). In a recent essay for The Century Foundation, where she is now a senior fellow, Brooks-LaSure used the 60th anniversary of enactment of those foundational insurance programs to help put their impact on individual Americans, the healthcare system and society at large in perspective. One prominent example is the desegregation of hospitals, which was achieved in part by withholding reimbursements for care unless facilities served Blacks as well as whites. Another is making it possible for more people with disabilities to live at home instead of in institutional settings. But as you'll hear in this probing Raise the Line conversation with host Lindsey Smith, Brooks-LaSure worries that many gains in coverage and other progress made over the years through Medicare, Medicaid and the Child Health Insurance Program (CHIP) are at risk because of a new federal law that calls for a trillion dollar decrease in spending, resulting in potentially millions of people losing their coverage, cuts to clinical staff and medical services, and the closure of hospitals and clinics, especially in rural areas. “Most rural hospitals in this country are incredibly dependent on both Medicare and Medicaid to keep their doors open and there's an estimate that over 300 hospitals will close as a result of this legislation, so that, I think, is a place of incredible nervousness.” Whether you are a patient, provider, policymaker or health system leader, this is a great opportunity to learn from an expert source about the range of potential impacts that will flow from changes to critically important insurance programs that provide coverage to 40% of adults and nearly 50% of children in the U.S. Mentioned in this episode:The Century FoundationEssay on 60th Anniversary of Medicare & Medicaid If you like this podcast, please share it on your social channels. You can also subscribe to the series and check out all of our episodes at www.osmosis.org/raisethelinepodcast

This week on the green scene, we are talking all about gene editing Pat and Ruth explore enhanced polo ponies, agricultural animals, and glow-in-the-dark plants for lighting.

It seems there are news stories every week about the accelerating pace of innovation in gene therapy, but only about 50 therapies have been approved so far by the US Food and Drug Administration. Our guest today, Dr. Bobby Gaspar, leads a UK-based biotech company, Orchard Therapeutics, that developed one of those treatments using gene-modified stem cells in your blood that self-renew, so a single administration can give you potentially a lifelong effect. “Our approach is about correcting those hematopoietic stem cells and allowing them to give rise to cells that can then correct the disease,” explains Dr. Gaspar.  The therapy in focus is lenmeldy, the first approved treatment for metachromatic leukodystrophy, also known as MLD, a devastating inherited disorder that affects roughly 600 children worldwide. But Dr. Gaspar is optimistic that learnings from Orchard's work on MLD could be useful in treating much more common disorders including frontotemporal dementia, Crohn's disease and others. This highly informative conversation with host Lindsey Smith also explores the importance of newborn screening, community collaboration in advancing clinical trials for rare diseases, and a future in which each gene therapy will be used as a tool for specific applications.  “There will be many gene therapies available, some of which will become the standard of care for certain diseases, but it won't be for every disease.”Mentioned in this episode:Orchard Therapeutics If you like this podcast, please share it on your social channels. You can also subscribe to the series and check out all of our episodes at www.osmosis.org/raisethelinepodcast

This Postmodern Realities episode is a conversation with JOURNAL author Rebekah Valerius about her  article, “G.K. Chesterton's ‘Orthodoxy' and Christian Enchantment”https://www.equip.org/articles/g-k-chestertons-orthodoxy-and-christian-enchantment/Related articles and podcasts by this author:Episode 422 Gene Editing and the Abolition of Man: A Reflection on ‘Upgrade' by Blake Crouch and the New Science of CRISPRGene Editing and the Abolition of Man: A Reflection on ‘Upgrade' by Blake Crouch and the New Science of CRISPREpisode 231 G. K. Chesterton and The Genius Of Job “G. K. Chesterton on the Book of Job”Episode 210: Bespoke Religiosity and the Rise of the Nones: a review of Strange Rites: New Religions for a Godless World by Tara Isabella BurtonBespoke Religiosity and the Rise of the Nones: a review of Strange Rites: New Religions for a Godless World by Tara Isabella BurtonEpisode 073: Hell and Kids Is It Abusive to Teach Children about HellJourney to the Bottom of the Glass: A Review of The Works of His Hands: A Scientist's Journey from Atheism to Faith by Sy Garte  Don't miss an episode; please subscribe to the Postmodern Realities podcast wherever you get your favorite podcasts. Please help spread the word about Postmodern Realities by giving us a rating and review when you subscribe to the podcast. The more ratings and reviews we have, the more new listeners can discover our content.

In this episode of Tiny Show and Tell Us, we talk about cute woolly mammoth mice and debate whether research to bring back extinct species (or their genes) is ethical or a good use of money and time. Then we discuss the best way to remove microplastics from water. We need your stories — they're what make these bonus episodes possible! Write in to tinymatters@acs.org *or fill out this form* with your favorite science fact or science news story for a chance to be featured.A transcript and references for this episode can be found at acs.org/tinymatters.See Privacy Policy at https://art19.com/privacy and California Privacy Notice at https://art19.com/privacy#do-not-sell-my-info.

War Room Bill Gates Says He Talked to Trump about Gene Editing Vaccines at Evil Villain Dinner Sky Pilot Radio The Soundtrack of your Life 60's thru the 80's

You are in for a dose of inspiration in this episode of Raise the Line as we introduce you to a rare disease patient who was a leading force in establishing the diagnosis for her own condition, who played a key role in launching the first phase three clinical trials for it, and who is now coordinating research into the disease and related disorders at one of the nation's top hospitals. Rebecca Salky, RN, was first afflicted at the age of four with MOGAD, an autoimmune disorder of the central nervous system that can cause paralysis, vision loss and seizures. In this fascinating conversation with host Lindsey Smith, Rebecca describes her long and challenging journey with MOGAD, her work at the Neuroimmunology Clinic and Research Lab at Massachusetts General Hospital, and the importance of finding a MOGAD community in her early twenties. “There's a sense of power and security when you have others on your side. You're not alone in this journey of the rare disease,” she explains. Be sure to stay tuned to learn about Rebecca's work in patient advocacy, her experience as a nurse, and the three things she thinks are missing in the care of rare disease patients as our Year of the Zebra series continues.Mentioned in this episode:The MOG ProjectNeuroimmunology Clinic & Research Lab at Mass General If you like this podcast, please share it on your social channels. You can also subscribe to the series and check out all of our episodes at www.osmosis.org/raisethelinepodcast

Walter Isaacson - bestselling biographer behind Musk, Einstein and Steve Jobs - in conversation with Evan Ratliff brings you behind the scenes of The Code Breaker: Jennifer Doudna, Gene Editing, and the Future of the Human Race. The story of the third great technological revolution in modern times. See omnystudio.com/listener for privacy information.

“You have to love what you do, especially in healthcare, and the earlier you find that, the better. So that's why I love to see HOSA helping young people find what it is that they want to do,” says Dr. David Kelly, a fellow in oculofacial surgery at University of California San Francisco and HOSA's board chair. You can still hear the excitement in Dr. Kelly's voice describing his earliest experiences with HOSA -- a student led organization with 300,000 plus members that prepares future health professionals to become leaders in international health – even though they happened sixteen years ago when he was a sophomore in highschool. Through hundreds of competitive events and hands-on projects, HOSA creates a framework for developing skills in communication, professionalism and leadership starting in middle school. Programs are offered throughout highschool and college as well, which Dr. Kelly took advantage of before becoming an active alumnus and joining the HOSA board as a way of giving back to an organization that has given so much to him. Since taking the reins as board chair last year, one key focus has been preparing to mark HOSA's 50th anniversary in 2026. Dr. Kelly sees the occasion as not only an opportunity to celebrate what HOSA has accomplished, but to ensure it is positioned to continue helping the healthcare industry tackle important challenges in the future. Examples include chronic workforce shortages and improving how clinicians communicate with patients and team members.  Join host Lindsey Smith on this uplifting Raise the Line episode for an optimistic look at the next generation of healthcare leaders.Mentioned in this episode:HOSAHOSA Alumni Registration If you like this podcast, please share it on your social channels. You can also subscribe to the series and check out all of our episodes at www.osmosis.org/raisethelinepodcast

Before CRISPR-based methods took center stage in the genome editing field, other bio-inspired tools such as zinc finger nucleases and Cre-lox system recombinases made genomic engineering possible. Some researchers are now looking back to these foundational technologies to improve upon and one-up the latest gene editing techniques.  In this episode, Deanna MacNeil from The Scientist spoke with Frank Buchholz, professor and head of the medical systems biology translational research group at the Technical University of Dresden's University Cancer Center, to explore how his laboratory designs programmable recombinases for high fidelity gene editing.   The Scientist Speaks is a podcast produced by The Scientist's Creative Services Team. Our podcast is by scientists and for scientists. We bring you the stories behind news-worthy molecular biology research. This episode is brought to you by Oxford Nanopore.

For UC Berkeley's Jennifer Doudna, the revolutionary discovery of CRISPR-Cas9 gene editing began 15 years ago with a meeting at the campus's Free Speech Movement Cafe. “This is a quintessential story about Berkeley,” begins Doudna, a professor of molecular and cell biology and of chemistry, in a lecture she gave on campus in April. “The research that I'll talk about today wouldn't have happened … if I had been working anywhere else. And that's because we have a really collaborative environment on our campus.”At the cafe, Doudna listened while a Berkeley colleague described a possible adaptive immune system in bacteria that helps them fight off viral infection. Doudna's lab went on to research the molecules involved, discovering a pathway that allows bacteria to "learn" about viruses, store the information and use it for protection.The scientists realized this same system could be used to trigger DNA repair in plant, animal and human cells, effectively allowing them to "rewrite the code of life." The seminal paper on CRISPR was published in 2012 by Doudna and her key collaborator, French microbiologist Emmanuelle Charpentier. The pair went on to win the Nobel Prize in Chemistry in 2020.In this Berkeley Talks episode, Doudna discusses how CRISPR can be used to correct disease-causing genetic mutations, the impact that it's already having on people's lives and where she sees the technology going in the future. “We're in an era of programmable genome editing,” she says. “It's really exciting to see all the possible applications of this. We know that it can be safe and effective to treat and even to potentially cure human disease, and we need to continue to advance the technology so that it can be deployed more widely.”Not only will that require continual activity on the science and technology front, she adds, but also in developing appropriate guidelines and regulations to ensure that CRISPR's applications move forward responsibly. Doudna's talk took place on April 4 as part of Brilliance of Berkeley, a course offered every spring by the College of Letters and Science that celebrates the campus's exceptional faculty and their accomplishments. Each week, students listen to two guest lectures by top Berkeley scholars from an array of fields, followed by a Q&A. Watch the video on the Brilliance of Berkeley YouTube page. Listen to the episode and read the transcript on UC Berkeley News (news.berkeley.edu/podcasts/berkeley-talks).Music by HoliznaCC0.Photo by Glenn Ramit/IGI. Hosted on Acast. See acast.com/privacy for more information.

“We've been able to show that even by 30 days of age, we can predict with some accuracy if a child is going to have a diagnosis of autism,” says Dr. Geraldine Dawson, sharing one of the recent advancements in early diagnosis being aided by artificial intelligence.  Dr. Dawson -- a leading scholar in the field and founding director of the Duke Center for Autism and Brain Development – explains that an AI examination of a child's pattern of visits to medical specialists in its very early life is an objective diagnostic tool that can supplement the current subjective reports from parents which vary in reliability. Another objective diagnostic tool in development uses a smartphone app developed at Duke that takes video of babies watching images and applies AI-aided Computer Vision Analysis to measure for signs of autism. This enlightening Raise the Line conversation with host Lindsey Smith is loaded with the latest understandings about Autism Spectrum Disorder including advancements in early therapeutic interventions, the interplay of genetic and environmental factors, and the role of the mother's health and exposures during pregnancy. You'll learn as well about what Dawson sees as necessary societal shifts in how autism is perceived, the numerous factors contributing to a near tripling of diagnoses over the past two decades, and how early intervention and informed advocacy can make a meaningful difference in the lives of countless families.Mentioned in this episode:Duke Center for Autism and Brain Development If you like this podcast, please share it on your social channels. You can also subscribe to the series and check out all of our episodes at www.osmosis.org/raisethelinepodcast

"Burnout and trauma are not mental illnesses. They live in your physiology. They live in your biology. They live very specifically in your nervous system,” Dr. Rola Hallam says with a conviction rooted in her own successful journey to overcome the effects of chronic stress she accumulated during many years on the frontlines of humanitarian crises in Syria and other conflict zones. Out of concern for the multitudes of health professionals who, like herself, spend years carrying the weight of their traumatic experiences without seeking help, or who pursue ineffective remedies for relieving it, Dr. Rola -- as she's known – has shifted her focus to being a trauma and burnout coach. Among her offerings is Beyond Burnout, a twelve-week program that includes multimedia content as well as live coaching and teaching about developing nervous system awareness and regulation. “Most wellness initiatives fail because they're not rewiring the nervous system to come out of survival mode and back into what is called the ventral-vagal state, which is our state of social connection and of healing and repair.” She also stresses that healing is not an individual pursuit, especially for providers who work in a relational field, and teaches about the benefits of borrowing from a colleagues' state of calm and offering them the same. Don't miss this insightful and giving conversation with host Lindsey Smith that covers Dr. Rola's wrenching experiences providing care in desperate conditions, the critically important distinction between empathy and compassion, and how empowering frontline workers to heal their trauma can uplift individuals and empower entire communities. Mentioned in this episode:Dr. Rola CoachingBeyond Burnout AssessmentCanDo - Humanitarian Aid If you like this podcast, please share it on your social channels. You can also subscribe to the series and check out all of our episodes at www.osmosis.org/raisethelinepodcast

“Seeing that you can get through the most difficult times in life, succeed, and then also return to your community and work in service to your community was a lesson that has stuck with me,” says Dr. Uche Blackstock, the Founder and CEO of Advancing Health Equity and our guest on this inspiring episode of Raise the Line with Osmosis from Elsevier. It was a lesson the Harvard-trained physician learned from her own mother – also a Harvard trained physician – who overcame poverty, sexism and racial bias to forge an inspiring path.  In her bestselling book, Legacy: A Black Physician Reckons with Racism in Medicine, Dr. Blackstock weaves her mother's remarkable story with her own and argues for systemic change in a healthcare system riddled with racially-biased practices and policies that impact patient outcomes. As she explains to host Lindsey Smith, Advancing Health Equity's work to drive measurable and sustainable change is focused on embedding equity as a core value in the leadership, strategy, and organizational practice of health systems. “We exist to challenge inequities, empower underrepresented communities, and help build a healthcare system where everyone can thrive.” Don't miss a thought-provoking conversation with a nationally respected voice that also addresses race correction factors that impact the care of Black patients, and the work required of health institutions to build trust in effected communities.Mentioned in this episode:Advancing Health EquityLegacy: A Black Physician Reckons with Racism in Medicine If you like this podcast, please share it on your social channels. You can also subscribe to the series and check out all of our episodes at www.osmosis.org/raisethelinepodcast

Dr. Hoffman continues his conversation with Zen Honeycutt, founder and executive director of Moms Across America and author of “Unstoppable: Transforming Sickness and Struggle into Triumph, Empowerment and a Celebration of Community.”