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Updated: Feb 12, 2026

The Rodent Model of Nonarteritic Anterior Ischemic Optic Neuropathy rNAION
Published on: November 20, 2016
Longterm Reversal of Severe Visual Loss by Mitochondrial Gene Transfer in a Mouse Model of Leber Hereditary Optic
Hong Yu1, Vittorio Porciatti1, Alfred Lewin2
1Bascom Palmer Eye Institute, University of Miami, Miller School of Medicine, Miami, FL, United States.
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.
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