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Towards a therapy for mitochondrial disease: an update

Published version
Peer-reviewed

Type

Article

Change log

Authors

Viscomi, CF 

Abstract

Preclinical work aimed at developing new therapies for mitochondrial diseases has recently given new hopes and opened unexpected perspectives for the patients affected by these pathologies. In contrast, only minor progresses have been achieved so far in the translation into the clinics. Many challenges are still ahead, including the need for a better characterization of the pharmacological effects of the different approaches and the design of appropriate clinical trials with robust outcome measures for this extremely heterogeneous, rare, and complex group of disorders. In this review, we will discuss the most important achievements and the major challenges in this very dynamic research field.

Description

Keywords

bypass therapy, gene therapy, mitochondrial biogenesis, mitochondrial dysfunction, rapamycin, Animals, Antioxidants, Clinical Trials as Topic, Diet, Ketogenic, Genetic Therapy, Humans, Hypoxia, Mechanistic Target of Rapamycin Complex 1, Mitochondria, Mitochondrial Diseases, Mutation, Phenotype, Reactive Oxygen Species, Sirolimus

Journal Title

Biochemical Society Transactions

Conference Name

Journal ISSN

1470-8752
1470-8752

Volume Title

46

Publisher

Portland Press
Sponsorship
European Research Council (322424)
Medical Research Council (MC_UU_00015/8)
Medical Research Council (MC_EX_MR/P007031/1)
MRC (MC_UU_00015/8)
Medical Research Council (MC_UU_00015/7)
Our work is supported by MRC (grants: MC_UU_00015/5 and MC_EX_MR/P007031/1) and ERC (grant FP7-322424)