Rescue of GM3 Synthase Deficiency by Spatially Controlled, rAAV-Mediated ST3GAL5 Delivery

Investigators showed that a first-generation rAAV-ST3GAL5 replacement vector using a ubiquitous promoter restored tissue ST3GAL5 expression and normalized cerebral gangliosides in patient-derived induced PSC neurons and brain tissue from St3gal5-KO mice but caused fatal hepatotoxicity when administered systemically.