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Chapter 7 Current and Future Prospects for the Treatment of Mitochondrial Disorders

DOI: 10.1016/s1877-3419(09)70066-2
  • Biology
  • Chemistry
  • Medicine


Publisher Summary This chapter describes the current and future prospects for the treatment of mitochondrial disorders. Whether the primary genetic lesion occurs in the mitochondrial genome (mitochondrial DNA [mtDNA]) or a nuclear gene, all mitochondrial disorders result from the progressive decline in the ability to supply cellular energy demands in the form of available adenosine triphosphate (ATP). The pivotal role occupied by the respiratory chain in cellular metabolism consequently poses acute difficulties in trying to overcome the respiratory defect. Biochemical strategies to increase the production of ATP have sought to bypass the block in electron transfer using artificial electron acceptors, enhance residual enzyme activity, or minimize the free radical-induced damage that occurs as a result of a defective respiratory chain. As mitochondrial dysfunction can present with problems in different systems, it is vital that diverse clinical problems be managed correctly through appropriate supportive medical care. Although these aids are no substitutes for treatment per se, particular supportive measures may significantly improve an individual's quality of life and help to counteract the progressive, disabling course of these disorders.

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