LHON Gene Therapy Vector Prevents Visual Loss and Optic Neuropathy Induced by G11778A Mutant Mitochondrial DNA: Biodistribution and Toxicology Profile [Eye Movements, Strabismus, Amblyopia, and Neuro-Ophthalmology]

Conclusions.
Allotopically expressed wild-type ND4 prevents the phenotype induced by G11778A mitochondrial DNA with a toxicology profile acceptable for testing in a phase I clinical trial.

Source: Investigative Ophthalmology - December 1, 2014 Category: Opthalmology Authors: Koilkonda, R., Yu, H., Talla, V., Porciatti, V., Feuer, W. J., Hauswirth, W. W., Chiodo, V., Erger, K. E., Boye, S. L., Lewin, A. S., Conlon, T. J., Renner, L., Neuringer, M., Detrisac, C., Guy, J. Tags: Eye Movements, Strabismus, Amblyopia, and Neuro-Ophthalmology Source Type: research