title: Toward a therapy for mitochondrial disease.
abstract: Mitochondrial disorders are a group of genetic diseases affecting the energy-converting process of oxidative phosphorylation. The extreme variability of symptoms, organ involvement, and clinical course represent a challenge to the development of effective therapeutic interventions. However, new possibilities have recently been emerging from studies in model organisms and awaiting verification in humans. I will discuss here the most promising experimental approaches and the challenges we face to translate them into the clinics. The current clinical trials will also be briefly reviewed.
authors.affiliation: MRC Mitochondrial Biology Unit, Wellcome Trust/MRC Building, Hills Road, Cambridge CB2 0XY, U.K.|Molecular Neurogenetics Unit, The Foundation 'Carlo Besta' Institute of Neurology, IRCCS, Milan 20133, Italy.
authors.forename: Carlo
authors.initials: C
authors.lastname: Viscomi
mesh_terms: D000818:Animals; D002986:Clinical Trials as Topic; D004272:DNA, Mitochondrial / Q000235:genetics* / Q000378:metabolism; D004734:Energy Metabolism / Q000235:genetics; D006801:Humans; D008928:Mitochondria / Q000235:genetics* / Q000378:metabolism; D028361:Mitochondrial Diseases / Q000235:genetics* / Q000378:metabolism / Q000628:therapy*; D017063:Outcome Assessment, Health Care; D017382:Reactive Oxygen Species / Q000378:metabolism; D057170:Translational Research, Biomedical / Q000379:methods
chemical_list: D004272:DNA, Mitochondrial; D017382:Reactive Oxygen Species
keywords: gene therapy; mitochondria; mitochondrial dysfunction; mouse models; therapeutics