Emerging concepts in the therapy of mitochondrial disease.

TitleEmerging concepts in the therapy of mitochondrial disease.
Publication TypeJournal Article
Year of Publication2015
AuthorsViscomi, C, Bottani, E, Zeviani, M
JournalBiochim Biophys Acta
Date Published2015 Jun-Jul
KeywordsElectron Transport, Humans, Mitochondrial Diseases, Oxidative Phosphorylation

Mitochondrial disorders are an important group of genetic conditions characterized by impaired oxidative phosphorylation. Mitochondrial disorders come with an impressive variability of symptoms, organ involvement, and clinical course, which considerably impact the quality of life and quite often shorten the lifespan expectancy. Although the last 20 years have witnessed an exponential increase in understanding the genetic and biochemical mechanisms leading to disease, this has not resulted in the development of effective therapeutic approaches, amenable of improving clinical course and outcome of these conditions to any significant extent. Therapeutic options for mitochondrial diseases still remain focused on supportive interventions aimed at relieving complications. However, new therapeutic strategies have recently been emerging, some of which have shown potential efficacy at the pre-clinical level. This review will present the state of the art on experimental therapy for mitochondrial disorders.

Alternate JournalBiochim. Biophys. Acta
Citation Key10.1016/j.bbabio.2015.03.001
PubMed ID25766847
Grant ListGGP11011 / / Telethon / Italy
MC_UP_1002/1 / / Medical Research Council / United Kingdom