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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Detalhes bibliográficos
Publicado no:Gene Ther
Main Authors: Di Meo, I, Marchet, S, Lamperti, C, Zeviani, M, Viscomi, C
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2017
Assuntos:
Short Communication
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5658670/
https://ncbi.nlm.nih.gov/pubmed/28753212
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gt.2017.53
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