Kyha D Williams

Works (1)

Updated: April 5th, 2024 11:49

2018 journal article

Long-term complications of glycogen storage disease type Ia in the canine model treated with gene replacement therapy

JOURNAL OF INHERITED METABOLIC DISEASE, 41(6), 965–976.

By: E. Brooks *, D. Landau*, J. Everitt*, T. Brown n, K. Grady*, L. Waskowicz*, C. Bass*, J. D'Angelo* ...

MeSH headings : Animals; Carcinoma, Hepatocellular / etiology; Dependovirus / genetics; Disease Models, Animal; Dogs; Female; Genetic Therapy; Genetic Vectors; Glucose-6-Phosphatase / genetics; Glucose-6-Phosphatase / metabolism; Glycogen Storage Disease Type I / complications; Glycogen Storage Disease Type I / therapy; Hypoglycemia / genetics; Hypoglycemia / metabolism; Liver / pathology; Liver Neoplasms / etiology; Male
topics (OpenAlex): Glycogen Storage Diseases and Myoclonus; Genetics and Neurodevelopmental Disorders; Oral and gingival health research
TL;DR: It is shown that the canine G SD Ia model demonstrates similar long-term complications as GSD Ia patients in spite of gene replacement therapy, and further development of gene therapy is needed to develop a more effective treatment to prevent long- term complications of GSDIa. (via Semantic Scholar)
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Source: Web Of Science
Added: February 4, 2019

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