Intravenous injection of AAVrh10-GALC after the neonatal period in twitcher mice results in significant expression in the central and peripheral nervous systems and improvement of clinical features
Keywords: سیستم عصبی پیرامونی; GLD; Globoid cell leukodystrophy; GALC; Galactocerebrosidase; twi; Twitcher; PND; Post-natal day; CNS; Central nervous system; PNS; Peripheral nervous system; HSCT; Hematopoietic stem cell transplantation; MLD; Metachromatic leukodystrophy; LFB/PAS; Luxol