Kanabus, M;
Heales, SJ;
Rahman, S;
(2014)
Development of Pharmacological Strategies for Mitochondrial Disorders.
Br J Pharmacol
, 171
(8)
pp. 1798-1817.
10.1111/bph.12456.
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Abstract
Mitochondrial diseases are an unusually genetically and phenotypically heterogeneous group of disorders, which are extremely challenging to treat. Currently, apart from supportive therapy, there are no effective treatments for the vast majority of mitochondrial diseases. Huge scientific effort however, is continuously put into understanding the mechanisms underlying mitochondrial disease pathology and developing potential treatments. To date a variety of treatments have been evaluated by randomised clinical trials, but unfortunately none of these has delivered breakthrough results. Increased understanding of mitochondrial pathways and the development of many animal models, some of which are accurate phenocopies of human diseases, are facilitating the discovery and evaluation of novel prospective treatments. Targeting reactive oxygen species has been a treatment of interest for many years, however only in recent years has it been possible to direct antioxidant delivery specifically into the mitochondria. Increasing mitochondrial biogenesis, whether by pharmacological approaches, dietary manipulation or exercise therapy is also currently an active area of research. Modulating mitochondrial dynamics and mitophagy and the mitochondrial membrane lipid milieu have also emerged as possible treatment strategies. Recent technological advances in gene therapy, including allotopic and transkingdom gene expression and mitochondrially-targeted transcription activator-like nucleases, have led to promising results in cell and animal models of mitochondrial diseases, but most of these techniques are still far from clinical applicability.
| Type: | Article |
|---|---|
| Title: | Development of Pharmacological Strategies for Mitochondrial Disorders. |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.1111/bph.12456 |
| Publisher version: | http://dx.doi.org/10.1111/bph.12456 |
| Additional information: | © 2013 The Authors. British Journal of Pharmacology published by John Wiley & Sons Ltd on behalf of The British Pharmacological Society. This is an open access article under the terms of the Creative Commons Attribution License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited. |
| Keywords: | Clinical trials for mitochondrial disorders, Gene therapy for mitochondrial diseases, Mitochondrial biogenesis, Mitochondrial diseases, Models for mitochondrial disorders, Nutritional and cofactor support in mitochondrial disorders, Treatments for mitochondrial disorders |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Genetics and Genomic Medicine Dept |
| URI: | https://discovery.ucl.ac.uk/id/eprint/1410315 |
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