Episode 200 -- Brian Kaspar PhD
Wednesday, April 10, 2019
\nBrian Kaspar (AveXis Inc) talks about the realities and promise of building a single-dose gene transfer therapy for treating the prime gene defect in spinal muscular atrophy (SMA) type I. He discusses his company\u2019s modified adeno-associated virus 9 approach to human gene therapy that is currently in highly promising clinical trials, for SMA, and in development for Rett syndrome and one variant of Amyotrophic Lateral Sclerosis.
\nDuration: 45 minutes
\nDiscussants:(in alphabetical order)
\nJenny Hsieh (Semmes Foundation Chair, UTSA)
\nSalma Quraishi (Res Asst Prof, UTSA)
\nCharles Wilson (Ewing Halsell Chair, UTSA)
\nacknowledgement: JM Tepper for original music.