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...

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Hlavní autoři: 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
Médium: Artigo
Jazyk:Inglês
Vydáno: Nature Publishing Group 2013
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Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC3522193/
https://ncbi.nlm.nih.gov/pubmed/22669418
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/ejhg.2012.112
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