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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.
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
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.
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Added: February 4, 2019
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