
Mitigation of cerebellar neuropathy in globoid cell leukodystrophy mice by AAV-mediated gene therapy
Keywords: AAV, adeno-associated virus; BDNF, brain-derived neurotrophic factor; BMT, bone marrow transplantation; CNS, central nervous system; CNTF, ciliary neurotrophic factor; GALC, galactocerebrosidase; GAPDH, glyceraldehyde 3-phosphate dehydrogenase; GDNF, glia