Single Systemic Administration of a Gene Therapy Leading to Disease Treatment in Metachromatic Leukodystrophy Arsa Knockout Mice

Single Systemic Administration of a Gene Therapy Leading to Disease Treatment in Metachromatic Leukodystrophy Arsa Knockout Mice

St. Martin T, Seabrook T, Gall K, et al. The Journal of Neuroscience 2023.