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Intravitreal delivery of AAV-NDI1 provides functional benefit in a murine model of Leber hereditary optic neuropathy
Leber hereditary optic neuropathy (LHON) is a mitochondrially inherited form of visual dysfunction caused by mutations in several genes encoding subunits of the mitochondrial respiratory NADH-ubiquinone oxidoreductase complex (complex I). Development of gene therapies for LHON has been impeded by ge...
Autores principales: | Chadderton, Naomi, Palfi, Arpad, Millington-Ward, Sophia, Gobbo, Oliverio, Overlack, Nora, Carrigan, Matthew, O'Reilly, Mary, Campbell, Matthew, Ehrhardt, Carsten, Wolfrum, Uwe, Humphries, Peter, Kenna, Paul F, Jane Farrar, G |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Nature Publishing Group
2013
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3522193/ https://www.ncbi.nlm.nih.gov/pubmed/22669418 http://dx.doi.org/10.1038/ejhg.2012.112 |
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