EP 208: Gene therapy at a crossroads: Successes, concerns, and the path forward

<p><strong>Summary: </strong></p><p>This week on The Genetics Podcast, Patrick Short takes a solo deep dive into the current state of gene therapy ahead of next week’s live recording at ASHG. He explores the promise and limitations of adeno-associated virus (AAV) delivery, examples of gene therapies for neuromuscular diseases, and the challenges of balancing safety, cost, and commercial viability in rare diseases. </p><p><strong>Show Notes: </strong></p><p><strong>0:00 </strong>Intro to The Genetics Podcast</p><p><strong>00:59</strong> Episode overview</p><p><strong>02:50</strong> Definition and scope of gene therapy </p><p><strong>04:50 </strong>Gene therapy delivery via adeno-associated virus (AAV) and associated challenges and advantages</p><p><strong>06:40 </strong>AAV-based gene therapy and other advanced therapies in spinal muscular atrophy (SMA)</p><p><strong>10:29 </strong>Recent safety concerns around Sarepta Therapeutics’ AAV-based gene therapy for Duchenne muscular dystrophy...