Mitochondrial encephalomyopathies: therapeutic approaches.
Therapy of mitochondrial encephalomyopathies (defined restrictively as defects of the mitochondrial respiratory chain) is woefully inadequate, despite great progress in our understanding of the molecular bases of these disorders. We review available and experimental therapeutic approaches, which fall into seven categories: (1) palliative therapy; (2) removal of noxious metabolites; (3) administration of artificial electron acceptors; (4) administration of metabolites and cofactors; (5) administration of oxygen radical scavengers; (6) gene therapy; and (7) genetic counseling. Progress in each of these approaches provides some glimmer of hope for the future, although much work remains to be done.
DiMauro
S
S
Department of Neurology, Columbia University College of Physicians and Surgeons, 630 West 168th Street, New York, NY 10032, USA.
Hirano
M
M
Schon
E A
EA
D004579:Electron Transport / Q000187:drug effects; D004734:Energy Metabolism / Q000187:drug effects; D016166:Free Radical Scavengers / Q000627:therapeutic use; D005817:Genetic Counseling; D015316:Genetic Therapy; D006801:Humans; D017237:Mitochondrial Encephalomyopathies / Q000235:genetics / Q000378:metabolism / Q000628:therapy*; D010166:Palliative Care
D016166:Free Radical Scavengers