Genetic Therapies Bootcamp: Foundations, Frontiers, and Clinical Engagement OnDemand-03-10 12-35 318-323 Yu Session 1 Overview of Genetic Therapies

03-10 12-35 318-323 Yu Session 1 Overview of Genetic Therapies

Video Transcription

Video Summary

Austin Larson and Tim Yu present a 35-year historical overview of genetic therapies, outlining their evolution, challenges, and ethical considerations. They distinguish key therapy types: gene replacement, gene editing, and RNA-based approaches. Landmark events include the first gene therapy trial in 1990, the tragic 1999 death of Jesse Gelsinger leading to improved safety and oversight, and milestones like the 2016 approval of nusinersen for spinal muscular atrophy (SMA). They highlight advances in delivery vectors, such as AAV9 for durable gene delivery and lipid nanoparticles enabling redosing and personalized treatment, exemplified by Baby KJ’s in vivo gene editing for CPS1 deficiency. The presenters emphasize the importance of understanding disease mechanisms, therapeutic windows, regulatory complexities, and the nuanced risk-benefit balance in genetic therapies. Ex vivo gene editing, as in sickle cell treatment, requires cell collection and chemotherapy, while in vivo strategies offer direct delivery. The talk sets the stage for deeper dives into these transformative, precise therapies reshaping treatment paradigms.

Keywords

genetic therapies

gene replacement

gene editing

RNA-based therapies

AAV9 delivery vector

lipid nanoparticles