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Systemic Delivery of AAV-Fdxr Mitigates the Phenotypes of Mitochondrial Disorders in Fdxr Mutant Mice

Gene therapy now provides a novel approach for treating inherited monogenetic disorders, including nuclear gene mutations associated with mitochondrial diseases. In this study, we have utilized a mouse model carrying a p.Arg389Gln mutation of the mitochondrial Ferredoxin Reductase gene (Fdxr) and tr...

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Detalles Bibliográficos
Autores principales:	Yang, Li, Slone, Jesse, Zou, Weiwei, Queme, Luis F., Jankowski, Michael P., Yin, Fei, Huang, Taosheng
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2020
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7488755/ https://www.ncbi.nlm.nih.gov/pubmed/32995353 http://dx.doi.org/10.1016/j.omtm.2020.05.021

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