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A Single Intravenous Injection of AAV-PHP.B-hNDUFS4 Ameliorates the Phenotype of Ndufs4(−/−) Mice
Leigh syndrome, or infantile necrotizing subacute encephalopathy (OMIM #256000), is one of the most common manifestations of mitochondrial dysfunction, due to mutations in more than 75 genes, with mutations in respiratory complex I subunits being the most common cause. In the present study, we used...
Gespeichert in:
| Veröffentlicht in: | Mol Ther Methods Clin Dev |
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| Hauptverfasser: | , , , , |
| Format: | Artigo |
| Sprache: | Inglês |
| Veröffentlicht: |
American Society of Gene & Cell Therapy
2020
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| Schlagworte: | |
| Online Zugang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7248291/ https://ncbi.nlm.nih.gov/pubmed/32478122 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2020.04.026 |
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