title: Treatment of Leber's Hereditary Optic Neuropathy.
abstract: Leber's hereditary optic neuropathy (LHON) is a rare mitochondrial disease of complex I of the respiratory chain. Patients typically present with subacute vision loss in one eye followed by the loss of vision in the second eye approximately 4-8 weeks later, ultimately leading to blindness. Therapeutic interventions have so far failed to prevent this vision loss; however, there has been recent interest in new managements for this prototypic mitochondrial disease. A review of the literature was performed and articles were identified using PubMed (the search terms LHON and treatment were used). These publications were paired to the clinical trials listed on the clinicaltrials.gov website. There are eighteen studies including 5 phase II/III clinical trials in LHON listed on the clinicaltrials.gov website and they vary from oral medication to eye drops and intravitreal injections. In this review, we discuss failed and active therapies and the future of clinical trials in LHON.
authors.affiliation: Ottawa Eye Institute Department of Ophthalmology, University of Ottawa The Ottawa General Hospital 501 Smyth Road Ottawa, Ontario,. Canada.
authors.forename: Rustum
authors.initials: R
authors.lastname: Karanjia
authors.affiliation: Doheny Eye Institute, Los Angeles, CA,. United States.
authors.forename: Jasdeep
authors.initials: J
authors.lastname: Chahal
authors.affiliation: Doheny Eye Institute, Los Angeles, CA,. United States.
authors.forename: Michael
authors.initials: M
authors.lastname: Ammar
authors.affiliation: Doheny Eye Center, Department of Ophthalmology, David Geffen School of Medicine at UCLA, Los Angeles, CA,. United States.
authors.forename: Alfredo A
authors.initials: AA
authors.lastname: Sadun
mesh_terms: D006801:Humans; D009883:Ophthalmic Solutions / Q000008:administration & dosage / Q000627:therapeutic use*; D029242:Optic Atrophy, Hereditary, Leber / Q000235:genetics / Q000378:metabolism / Q000628:therapy*; D017382:Reactive Oxygen Species / Q000378:metabolism; D033581:Stem Cell Transplantation; D013234:Stem Cells
chemical_list: D009883:Ophthalmic Solutions; D017382:Reactive Oxygen Species
keywords: LHON; Mitochondria; gene therapy; optic neuropathy; retinal ganglion cell; treatment