A critical approach to the therapy of mitochondrial respiratory chain and oxidative phosphorylation diseases

BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE

卷:1792

Review

DiMauro, Salvatore³  Rustin, Pierre^1,2 

[1] Hop Robert Debre, INSERM, U676, F-75019 Paris, France

[2] Univ Paris 07, Fac Med Denis Diderot, IFR02, F-75005 Paris, France

[3] Columbia Univ, Med Ctr, Dept Neurol, New York, NY 10032 USA

关键词: Mitochondrial disease;    Respiratory chain;    Coenzyme Q10;    Therapy;   

DOI  :  10.1016/j.bbadis.2008.10.015

来源: Elsevier

【 摘 要 】

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. (C) 2008 Elsevier B.V. All rights reserved.

【 授权许可】

Free