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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)...
Autores principales: | 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. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Society of Gene & Cell Therapy
2019
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6909089/ https://www.ncbi.nlm.nih.gov/pubmed/31890731 http://dx.doi.org/10.1016/j.omtm.2019.10.016 |
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