Neuroscientists Talk Shop

Wednesday, April 10, 2019 Brian 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’s 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. Duration: 45 minutes Discussants:(in alphabetical order) Jenny Hsieh (Semmes Foundation Chair, UTSA) Salma Quraishi (Res Asst Prof, UTSA) Charles Wilson (Ewing Halsell Chair, UTSA) acknowledgement: JM Tepper for original music.