Experimental strategies towards treating mitochondrial DNA disorders.
An extensive range of molecular defects have been identified in the human mitochondrial genome (mtDNA), causing a range of clinical phenotypes characterized by mitochondrial respiratory chain dysfunction. Sadly, given the complexities of mitochondrial genetics, there are no available cures for mtDNA disorders. In this review, we consider experimental, genetic-based strategies that have been or are being explored towards developing treatments, focussing on two specific areas which we are actively pursuing--assessing the benefit of exercise training for patients with mtDNA defects, and the prevention of mtDNA disease transmission.
Gardner
Julie L
JL
Mitochondrial Research Group, School of Neurology, Neurobiology and Psychiatry, The Medical School, Newcastle University, Newcastle upon Tyne, NE2 4HH, UK.
Craven
Lyndsey
L
Turnbull
Douglass M
DM
Taylor
Robert W
RW
D000818:Animals; D004272:DNA, Mitochondrial / Q000235:genetics; D005081:Exercise Therapy / Q000379:methods; D015316:Genetic Therapy / Q000379:methods; D018095:Germ-Line Mutation; D006801:Humans; D018445:Infectious Disease Transmission, Vertical / Q000517:prevention & control; D028361:Mitochondrial Diseases / Q000235:genetics / Q000628:therapy*; D035703:Therapies, Investigational / Q000379:methods*
D004272:DNA, Mitochondrial