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- Title
AAV-mediated gene delivery in adult GM1-gangliosidosis mice corrects lysosomal storage in CNS and improves survival.
- Authors
Baek, Rena C; Broekman, Marike L D; Leroy, Stanley G; Tierney, Laryssa A; Sandberg, Michael A; d'Azzo, Alessandra; Seyfried, Thomas N; Sena-Esteves, Miguel
- Abstract
GM1-gangliosidosis is a glycosphingolipid (GSL) lysosomal storage disease caused by a genetic deficiency of acid β-galactosidase (βgal), which results in the accumulation of GM1-ganglioside and its asialo-form (GA1) primarily in the CNS. Age of onset ranges from infancy to adulthood, and excessive ganglioside accumulation produces progressive neurodegeneration and psychomotor retardation in humans. Currently, there are no effective therapies for the treatment of GM1-gangliosidosis.
- Publication
PloS one, 2010, Vol 5, Issue 10, pe13468
- ISSN
1932-6203
- Publication type
Journal Article
- DOI
10.1371/journal.pone.0013468