In these trials, participants affected by LHON with the G11778A mutation will have a virus expressing the functional version of ND4—the gene mutated in this variant of LHON—injected into one eye. A sham injection will be administered to the other eye for comparison. It is hypothesized that introduction of the viral vector may be able to rescue the function of the mutant gene. Preliminary results have demonstrated tolerability of the injections in a small number of subjects.Stealth BioTherapeutics is investigating the use of elamipretide (MTP-131), a mitochondrial protective agent, as a therapy for LHON. Elamipretide helps stabilize cardiolipin—an important component of mitochondrial inner membranes—and has been shown to reduce damaging reactive oxygen species in animal models. See also
Amaurosis
Dominant optic atrophy
Glaucoma
Ischemic optic neuropathy
Optic atrophy
Toxic and nutritional optic neuropathy

References
Further reading
Lebers hereditary optic neuropathy at NLM Genetics Home Reference
Kerrison JB, Newman NJ (1997). "Clinical spectrum of Lebers hereditary optic neuropathy" (IFOND reprints). Clin. Neurosci. 4 (5): 295–301. PMID 9292259. Carelli V, Ross-Cisneros FN, Sadun AA (January 2004). "Mitochondrial dysfunction as a cause of optic neuropathies". Prog Retin Eye Res. 23 (1): 53–89. doi:10.1016/j.preteyeres.2003.10.003. PMID 14766317. S2CID 15862778. External links

NCBI Genetic Testing Registry
Journal of Molecular and Cellular Cardiology. 33 (4): 655–670. doi:10.1006/jmcc.2001.1340. ISSN 0022-2828. PMC 2901497. PMID 11273720. Acton, Q. Ashton (2013). Advances in Heart Research and Application: 2013 Edition. Scholarly Editions. ISBN 978-1-481-68280-0. Towbin, JA (2014). "Inherited cardiomyopathies". Circulation Journal. 78 (10): 2347–56. doi:10.1253/circj.cj-14-0893. ISSN 1347-4820. PMC 4467885. PMID 25186923. Maron, Barry J.; Udelson, James E.; Bonow, Robert O.; Nishimura, Rick A.; Ackerman, Michael J.; Estes, N. A. Mark; Cooper, Leslie T.; Link, Mark S.; Maron, Martin S. (1 December 2015). "Eligibility and Disqualification Recommendations for Competitive Athletes With Cardiovascular Abnormalities: Task Force 3: Hypertrophic Cardiomyopathy, Arrhythmogenic Right Ventricular Cardiomyopathy and Other Cardiomyopathies, and Myocarditis: A Scientific Statement From the American Heart Association and American College of Cardiology". Circulation. 132 (22): e273–280. doi:10.1161/CIR.0000000000000239. ISSN 1524-4539. PMID 26621644. S2CID 207639288. External links
Cardiomyopathy at Curlie