Longterm Reversal of Severe Visual Loss by Mitochondrial Gene Transfer in a Mouse Model of Leber Hereditary Optic Neuropathy

Hong Yu, Vittorio Porciatti, Alfred Lewin, William Hauswirth, John Guy

Research output: Contribution to journalArticle

5 Scopus citations

Abstract

In many human disorders mitochondrial dysfunction is central to degeneration of retinal ganglion cells. As these cells do not regenerate, vision is irreversibly lost. Here we show reversal of visual dysfunction by a mitochondrially targeted adeno associated virus in transgenic mice harboring a G11778A mutation in the ND4 subunit of complex I persists longterm and it is associated with reduced loss of RGCs and their axons, improved oxidative phosphorylation, persistence of transferred ND4 DNA and transcription of ND4 mRNA.

Original languageEnglish (US)
Article number5587
JournalScientific reports
Volume8
Issue number1
DOIs
StatePublished - Dec 1 2018

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