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.Keywords: Mitochondria, retinal ganglion cell, LHON, optic neuropathy, treatment, gene therapy.
Current Pharmaceutical Design
Title:Treatment of Leber’s Hereditary Optic Neuropathy
Volume: 23 Issue: 4
Author(s): Rustum Karanjia, Jasdeep Chahal, Michael Ammar and Alfredo A. Sadun
Affiliation:
Keywords: Mitochondria, retinal ganglion cell, LHON, optic neuropathy, treatment, gene therapy.
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.Export Options
About this article
Cite this article as:
Karanjia Rustum, Chahal Jasdeep, Ammar Michael and Sadun A. Alfredo, Treatment of Leber’s Hereditary Optic Neuropathy, Current Pharmaceutical Design 2017; 23 (4) . https://dx.doi.org/10.2174/1381612823666170125164856
DOI https://dx.doi.org/10.2174/1381612823666170125164856 |
Print ISSN 1381-6128 |
Publisher Name Bentham Science Publisher |
Online ISSN 1873-4286 |
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