Towards a therapy for mitochondrial disease: an update
Published version
Peer-reviewed
Repository URI
Repository DOI
Change log
Authors
Viscomi, CF
Garone, Caterina https://orcid.org/0000-0003-4928-1037
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
1470-8752
Volume Title
46
Publisher
Portland Press
Publisher DOI
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)
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)