title: Strategies for treating disorders of the mitochondrial genome.
abstract: Defects of the mitochondrial genome are a significant cause of disease. Patients suffer from a wide variety of clinical presentations, ranging from fatal infantile disease to mild muscle weakness. Most disorders, however, are characterized by inexorable progression. As mutations often cause defects in several components of the complexes that couple oxidative phosphorylation, this terminal state of oxidative metabolism cannot be readily bypassed by dietary means, leading to the search for novel therapies. In this article, we present the theory behind several concepts and report progress. We also discuss some of the recent difficulties encountered in the progress towards an antigenomc approach to treating mtDNA disorders.
authors.affiliation: Mitochondrial Research Group, The Medical School, University of Newcastle upon Tyne, Framlington Place, Newcastle upon Tyne, NE2 4HH, UK.
authors.forename: Paul M
authors.initials: PM
authors.lastname: Smith
authors.forename: Günther F
authors.initials: GF
authors.lastname: Ross
authors.forename: Robert W
authors.initials: RW
authors.lastname: Taylor
authors.forename: Douglass M
authors.initials: DM
authors.lastname: Turnbull
authors.forename: Robert N
authors.initials: RN
authors.lastname: Lightowlers
mesh_terms: D001692:Biological Transport; D004272:DNA, Mitochondrial / Q000235:genetics / Q000378:metabolism; D005786:Gene Expression Regulation; D015316:Genetic Therapy / Q000379:methods* / Q000639:trends; D015894:Genome, Human; D006801:Humans; D028361:Mitochondrial Diseases / Q000235:genetics* / Q000628:therapy*; D024101:Mitochondrial Proteins / Q000235:genetics / Q000378:metabolism; D009154:Mutation; D012343:RNA, Transfer / Q000235:genetics / Q000378:metabolism
chemical_list: D004272:DNA, Mitochondrial; D024101:Mitochondrial Proteins; D012343:RNA, Transfer