Pathogenesis of Hepatic Tumors following Gene Therapy in Murine and Canine Models of Glycogen Storage Disease

Glycogen storage disease type Ia (GSD Ia) is caused by mutations in the glucose-6-phosphatase (G6Pase) catalytic subunit gene (G6PC). GSD Ia complications include hepatocellular adenomas (HCA) with a risk for hepatocellular carcinoma (HCC) formation. Genome editing with adeno-associated virus (AAV)...

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Publicado no:Mol Ther Methods Clin Dev
Main Authors: Kang, Hye-Ri, Gjorgjieva, Monika, Smith, Stephanie N., Brooks, Elizabeth D., Chen, Zelin, Burgess, Shawn M., Chandler, Randy J., Waskowicz, Lauren R., Grady, Kylie M., Li, Songtao, Mithieux, Gilles, Venditti, Charles P., Rajas, Fabienne, Koeberl, Dwight D.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2019
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6909089/
https://ncbi.nlm.nih.gov/pubmed/31890731
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2019.10.016
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