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AAV9-based gene therapy partially ameliorates the clinical phenotype of a mouse model of Leigh syndrome

Leigh syndrome (LS) is the most common infantile mitochondrial encephalopathy. No treatment is currently available for this condition. Mice lacking Ndufs4, encoding NADH: ubiquinone oxidoreductase iron-sulfur protein 4 (NDUFS4) recapitulates the main findings of complex I (cI)-related LS, including...

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Detalles Bibliográficos
Autores principales:	Di Meo, I, Marchet, S, Lamperti, C, Zeviani, M, Viscomi, C
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group 2017
Materias:	Short Communication
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5658670/ https://www.ncbi.nlm.nih.gov/pubmed/28753212 http://dx.doi.org/10.1038/gt.2017.53

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