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Review
. 2016 Oct 15;44(5):1483-1490.
doi: 10.1042/BST20160085.

Toward a therapy for mitochondrial disease

Carlo Viscomi  1   2
Affiliations
Review

Toward a therapy for mitochondrial disease

Carlo Viscomi. Biochem Soc Trans. .

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.

Keywords: gene therapy; mitochondria; mitochondrial dysfunction; mouse models; therapeutics.

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