Gene delivery improvement for treating the lysosomal storage disorder metachromatic leukodystrophy.
Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA.
Laura
L
Ferraiuolo
Brian K
BK
Kaspar
D000818:Animals; D001921:Brain / Q000378:metabolism*; D000229:Dependovirus / Q000235:genetics*; D006801:Humans; D007966:Leukodystrophy, Metachromatic / Q000235:genetics* / Q000628:therapy*; D009836:Oligodendroglia / Q000378:metabolism*