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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Bibliografiske detaljer
Udgivet i:Mol Ther Methods Clin Dev
Main Authors: Yang, Li, Slone, Jesse, Zou, Weiwei, Queme, Luis F., Jankowski, Michael P., Yin, Fei, Huang, Taosheng
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Gene & Cell Therapy 2020
Fag:
Original Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7488755/
https://ncbi.nlm.nih.gov/pubmed/32995353
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2020.05.021
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