期刊
BIOCHIMICA ET BIOPHYSICA ACTA-BIOENERGETICS
卷 1847, 期 6-7, 页码 544-557出版社
ELSEVIER
DOI: 10.1016/j.bbabio.2015.03.001
关键词
Mitochondrion; Experimental therapy; Oxidative phosphorilation; Mitochondrial respiratory chain; Mitochondrial disease; Animal model
资金
- MRC [MC_UP_1002/1]
- Pierfranco and Luisa Mariani Foundation Italy [Ricerca2000]
- Telethon-Italy [GPP10005, GGP11011]
- Cariplo [2011-0526]
- ERC [FP7-322424]
- Italian Ministry of Health [GR-2010-2306-756]
- MRC [MC_UP_1002/1] Funding Source: UKRI
- Medical Research Council [MC_UP_1002/1] Funding Source: researchfish
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. Crown Copyright (C) 2015 Published by Elsevier B.V.
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