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Systemic administration of AAV-Slc25a46 mitigates mitochondrial neuropathy in Slc25a46(−/−) mice

Mitochondrial disorders are the result of nuclear and mitochondrial DNA mutations that affect multiple organs, with the central and peripheral nervous system often affected. Currently, there is no cure for mitochondrial disorders. Currently, gene therapy offers a novel approach for treating monogene...

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Detalles Bibliográficos
Publicado en:Hum Mol Genet
Main Authors: Yang, Li, Slone, Jesse, Li, Zhuo, Lou, Xiaoting, Hu, Yueh-Chiang, Queme, Luis F, Jankowski, Michael P, Huang, Taosheng
Formato: Artigo
Idioma:Inglês
Publicado: Oxford University Press 2020
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Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC7068115/
https://ncbi.nlm.nih.gov/pubmed/31943007
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddz277
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