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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Bibliographic Details
Main Authors: 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
Format: Online
Language:English
Published: Nature Publishing Group 2013
Online Access:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3522193/