SMN2 and spinal muscular atrophy, type 1: The extraordinary results with viral-mediated gene transfer in SMA mice and in nonhuman primates recently led AveXis to start a phase I clinical trial at Ohio State University (OSU) to assess safety and tolerability of intravenously injected scAAV9-SMN (referred to as “chariSMA”) in patients with SMA type 1 (Fig. 2).