AAV-Mediated Gene Delivery in Adult GM1-Gangliosidosis Mice Corrects Lysosomal Storage in CNS and Improves Survival

BACKGROUND: 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...

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Bibliografische gegevens
Hoofdauteurs: 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
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: Public Library of Science 2010
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Research Article
Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC2956705/
https://ncbi.nlm.nih.gov/pubmed/20976108
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0013468
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