Article
AAV2/5 vector expressing galactocerebrosidase ameliorates CNS disease in the murine model of globoid-cell leukodystrophy more efficiently than AAV2.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 Sept 2005
Lin Darshong, Fantz Corinne R, Levy Beth, Rafi Mohammad A, Vogler Carole, Wenger David A, Sands Mark S
Abstract excerpt
Globoid-cell leukodystrophy (GLD) is an autosomal recessive lysosomal storage disorder caused by mutations in the galactosylceramidase (GALC) gene. Infantile GLD has a lethal course with severe cerebral demyelination that progresses to death by 2 years of age. In the current study twitcher mice, an authentic murine model of infantile GLD, were given intracranial injections of either recombinant adeno-associated...
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