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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Bibliografische gegevens
Gepubliceerd in:Hum Mol Genet
Hoofdauteurs: Yang, Li, Slone, Jesse, Li, Zhuo, Lou, Xiaoting, Hu, Yueh-Chiang, Queme, Luis F, Jankowski, Michael P, Huang, Taosheng
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: Oxford University Press 2020
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General Article ONE
Online toegang: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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