Increasing mtDNA levels as therapy for mitochondrial optic neuropathies. Issue 3 (March 2018)

• Record Type: Journal Article
• Authors: Ruiz-Pesini, Eduardo; Emperador, Sonia; López-Gallardo, Ester; Hernández-Ainsa, Carmen; Montoya, Julio
• Is Part Of: Drug discovery today. Volume 23:Issue 3(2018)
• Page Start: 493
• Page End: 498
• Publication Date: 2018-03
• Subjects: Drugs -- Design -- Periodicals; Drugs -- Research -- Periodicals; 615.1
• DOI: 10.1016/j.drudis.2018.01.031
• Languages: English
• ISSNs: 1359-6446

Abstract

Highlights: Leber hereditary optic neuropathy (LHON) is a mitochondrial optic neuropathy (MON). Primary open angle glaucoma (POAG) can be a MON. LHON and POAG risk factors include reduced mitochondrial DNA (mtDNA) quantity. Increasing mtDNA levels is a potential MON therapeutic option. Abstract : Leber hereditary optic neuropathy (LHON) is a rare, inherited mitochondrial disease. No treatment has shown a clear-cut benefit on a clinically meaningful end-point. Primary open-angle glaucoma (POAG) is a frequent, acquired optic neuropathy. Lowering intraocular pressure (IOP) reduces disease progression. However, current methods to decelerate this progression are recognized as being inadequate. Therefore, there is a clear need to look for new therapeutic approaches. The growing evidence indicates that POAG can also be a mitochondrial optic neuropathy (MON). Several risk elements are common for both diseases and all of them decrease mitochondrial (mt)DNA content. Based on these susceptibility factors and their molecular mechanism, we suggest herein pharmacological therapies targeted to increase mtDNA levels, oxidative phosphorylation (OXPHOS) capability, and mitochondrial energy production as treatments for MONs.