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 Amyotropic 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.