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.
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.
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open access via europepmc.org (repository)
UN Sustainable Development Goal Categories
3. Good Health and Well-being
(Web of Science; OpenAlex)
Source: Web Of Science
Added: August 6, 2018
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