Medicinal and Genetic Approaches to the Treatment of Mitochondrial Disease

Eric   A.   Schon; Salvatore      DiMauro

doi:10.2174/0929867033456503

Abstract

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, therapy, respiratory chain

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Current Medicinal Chemistry

Title: Medicinal and Genetic Approaches to the Treatment of Mitochondrial Disease

Volume: 10 Issue: 23

Author(s): Eric A. Schon and Salvatore DiMauro

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

Abstract: 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.

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Cite this article as:

Schon A. Eric and DiMauro Salvatore, Medicinal and Genetic Approaches to the Treatment of Mitochondrial Disease, Current Medicinal Chemistry 2003; 10 (23) . https://dx.doi.org/10.2174/0929867033456503

DOI https://dx.doi.org/10.2174/0929867033456503	Print ISSN 0929-8673
Publisher Name Bentham Science Publisher	Online ISSN 1875-533X

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