Long-Term Efficacy Following Readministration of an Adeno-Associated Virus Vector in Dogs with Glycogen Storage Disease Type Ia
Demaster, A., Luo, X., Curtis, S., Williams, K. D., Landau, D. J., Drake, E. J., … Koeberl, D. D. (2011, December 20). Human Gene Therapy.
By:
A. Demaster, X. Luo, S. Curtis, K. Williams, D. Landau, E. Drake, D. Kozink n, A. Bird ..., B. Crane n, F. Sun, C. Pinto n, T. Brown n, A. Kemper & D. Koeberl
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
topics (OpenAlex): Virus-based gene therapy research; Biochemical and Molecular Research; Neurogenetic and Muscular Disorders Research
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.
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