Towards a therapy for mitochondrial disease: an update.

TitleTowards a therapy for mitochondrial disease: an update.
Publication TypeJournal Article
Year of Publication2018
AuthorsGarone, C, Viscomi, C
JournalBiochem Soc Trans
Volume46
Issue5
Pagination1247-1261
Date Published2018 10 19
ISSN1470-8752
KeywordsAnimals, 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
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.

DOI10.1042/BST20180134
Alternate JournalBiochem. Soc. Trans.
Citation Key10.1042/BST20180134
PubMed ID30301846
PubMed Central IDPMC6195631
Grant ListMC_UU_00015/5 / / Medical Research Council / United Kingdom
MC_EX_MR/P007031/1] / / Medical Research Council / United Kingdom