A critical approach to the therapy of mitochondrial respiratory chain and oxidative phosphorylation diseases.
Taking advantage of a series of questions raised by an association of patients with mitochondrial disease, this review, after a brief overview of basic concepts of mitochondrial bioenergetics and genetics, discusses the pros and cons of a number of practical options in the field of mitochondrial therapy. This makes it clear that, in contrast to the spectacular progress in our understanding of the biochemical and molecular bases of the mitochondrial diseases defined restrictively as disorders due to defects in the mitochondrial respiratory chain, we are still extremely limited in our ability to treat these conditions. We finally discussed the emerging genetic-based strategies that show some promise, even if much work remains to be done.
Dimauro
Salvatore
S
Department of Neurology, Columbia University Medical Center, 313 Russ Berrie Medical Science Pavilion, 1150 St. Nicholas Avenue, New York, NY 10032, USA.
Rustin
Pierre
P
D004579:Electron Transport / Q000502:physiology*; D006801:Humans; D028361:Mitochondrial Diseases / Q000378:metabolism / Q000473:pathology / Q000628:therapy*; D010085:Oxidative Phosphorylation