Bayley Crane

Works (1)

Updated: July 5th, 2023 15:46

2011 journal article

Long-Term Efficacy Following Readministration of an Adeno-Associated Virus Vector in Dogs with Glycogen Storage Disease Type Ia

HUMAN GENE THERAPY, 23(4), 407–418.

By: A. Demaster*, X. Luo *, S. Curtis *, K. Williams*, D. Landau*, E. Drake*, D. Kozink n, A. Bird* ...

MeSH headings : Animals; Dependovirus / genetics; Dogs; Genetic Therapy; Genetic Vectors; Glucose-6-Phosphatase / genetics; Glucose-6-Phosphatase / metabolism; Glycogen Storage Disease Type I / genetics; Glycogen Storage Disease Type I / metabolism; Glycogen Storage Disease Type I / therapy; Hypoglycemia / genetics; Hypoglycemia / metabolism; Hypoglycemia / therapy; Liver / metabolism
TL;DR: Preclinical data support the further translation of AAV vector-mediated gene therapy in GSD-Ia and support the demonstrated risk for recurrent hypoglycemia and mortality from waning efficacy of the AAV2/9 vector. (via Semantic Scholar)
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