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Lookup NU author(s): Dr Oliver Russell,
Professor Grainne Gorman,
Professor Robert Lightowlers,
Emeritus Professor Doug Turnbull
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© 2020 Russell et al. review recent progress in the varied approaches to treat and prevent mitochondrial disease, including small-molecule-based approaches to improve mitochondrial function or turnover, manipulation of the mitochondrial genome, and mitochondrial replacement therapy.© 2020Mitochondrial diseases are clinically heterogeneous disorders caused by a wide spectrum of mutations in genes encoded by either the nuclear or the mitochondrial genome. Treatments for mitochondrial diseases are currently focused on symptomatic management rather than improving the biochemical defect caused by a particular mutation. This review focuses on the latest advances in the development of treatments for mitochondrial disease, both small molecules and gene therapies, as well as methods to prevent transmission of mitochondrial disease through the germline.
Author(s): Russell OM, Gorman GS, Lightowlers RN, Turnbull DM
Publication type: Review
Publication status: Published
Print publication date: 02/04/2020
Online publication date: 26/03/2020
Acceptance date: 02/04/2018
ISSN (print): 0092-8674
ISSN (electronic): 1097-4172
Publisher: Cell Press