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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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Dettagli Bibliografici
Pubblicato in:Hum Mol Genet
Autori principali: Yang, Li, Slone, Jesse, Li, Zhuo, Lou, Xiaoting, Hu, Yueh-Chiang, Queme, Luis F, Jankowski, Michael P, Huang, Taosheng
Natura: Artigo
Lingua:Inglês
Pubblicazione: Oxford University Press 2020
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Accesso online: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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