Gene Therapy: Targets and Therapeutics

This lecture covers gene therapy targets, including receptors, enzymes, ion channels, and protein therapeutics. It discusses gene therapy principles, delivery challenges, and successes in treating monogenic diseases like spinal muscular atrophy. The use of RNA-based therapeutics, such as antisense inhibitors and RNA interference, is also explored, with examples like Nusinersen for SMA and Patisiran for hereditary transthyretin-mediated amyloidosis. The lecture delves into the history of gene therapy, from the first cure in a mouse to recent FDA-approved treatments like Luxturna and Kymriah. It highlights the challenges and advancements in gene therapy, including the development of CAR T cell therapy and CRISPR-based treatments for genetic disorders like Leber congenital amaurosis.