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Updated: July 5th, 2023 15:53

2008 journal article

AAV vector-mediated reversal of hypoglycemia in canine and murine glycogen storage disease type Ia

MOLECULAR THERAPY, 16(4), 665–672.

By: D. Koeberl*, C. Pinto n, B. Sun*, S. Li*, D. Kozink n, D. Benjamin*, A. Demaster n, M. Kruse n ...

MeSH headings : Animals; Dependovirus / genetics; Disease Models, Animal; Dogs; Genetic Therapy; Genetic Vectors; Glucose-6-Phosphatase / biosynthesis; Glucose-6-Phosphatase / genetics; Glycogen Storage Disease Type I / enzymology; Glycogen Storage Disease Type I / therapy; Humans; Hypoglycemia / enzymology; Hypoglycemia / therapy; Liver Glycogen / metabolism; Mice; Mice, Knockout
TL;DR: The AAV2/8 vector uniquely prevented hypoglycemia during fasting and fully corrected liver G6Pase deficiency in GSD-Ia mice and dogs, and support the further preclinical development of AAV vector-mediated gene therapy for GSD -Ia. (via Semantic Scholar)
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