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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...

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Detalles Bibliográficos
Autores principales: Silva-Pinheiro, Pedro, Cerutti, Raffaele, Luna-Sanchez, Marta, Zeviani, Massimo, Viscomi, Carlo
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7248291/
https://www.ncbi.nlm.nih.gov/pubmed/32478122
http://dx.doi.org/10.1016/j.omtm.2020.04.026

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