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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Shranjeno v:
Bibliografske podrobnosti
izdano v:Hum Mol Genet
Main Authors: Yang, Li, Slone, Jesse, Li, Zhuo, Lou, Xiaoting, Hu, Yueh-Chiang, Queme, Luis F, Jankowski, Michael P, Huang, Taosheng
Format: Artigo
Jezik:Inglês
Izdano: Oxford University Press 2020
Teme:
General Article ONE
Online dostop: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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