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Publication : Emerging concepts in the therapy of mitochondrial disease.

First Author  Viscomi C Year  2015
Journal  Biochim Biophys Acta Volume  1847
Issue  6-7 Pages  544-57
PubMed ID  25766847 Mgi Jnum  J:254064
Mgi Id  MGI:6100217 Doi  10.1016/j.bbabio.2015.03.001
Citation  Viscomi C, et al. (2015) Emerging concepts in the therapy of mitochondrial disease. Biochim Biophys Acta 1847(6-7):544-57
abstractText  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.
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