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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Gorde:
Xehetasun bibliografikoak
Argitaratua izan da:Mol Ther Methods Clin Dev
Egile Nagusiak: 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.
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: American Society of Gene & Cell Therapy 2019
Gaiak:
Article
Sarrera elektronikoa: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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