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Medicinal and Genetic Approaches to the Treatment of Mitochondrial Disease

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Although great progress has been made in our understanding of the molecular bases of mitochondrial disorders due to defects in the respiratory chain, little exists in the way of rational therapy. Possible therapeutic approaches include: palliative therapy; removal of noxious metabolites; administration of artificial electron acceptors, metabolites, and free radical scavengers; genetic counseling; and gene therapy. There has been progress with each of these approaches, although much work remains to be done. Finally, a novel approach to treating a specific mitochondrial disorder, MELAS, is presented.

Keywords: melas, mtdna; nitric oxide; nitroglycerin; oxidative phosphorylation; respiratory chain; therapy

Document Type: Review Article


Affiliations: Columbia University, Department of Neurology - Room 4-431, 630 West 168th Street, NY New York, 10032, USA.

Publication date: 2003-12-01

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  • Current Medicinal Chemistry covers all the latest and outstanding developments in medicinal chemistry and rational drug design. Each issue contains a series of timely in-depth reviews written by leaders in the field covering a range of the current topics in medicinal chemistry. Current Medicinal Chemistry is an essential journal for every medicinal chemist who wishes to be kept informed and up-to-date with the latest and most important developments.
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