Approaches and limitations to gene therapy for mitochondrial diseases.
Mitochondrial dysfunction may be caused by mutations in either the nuclear and/or the mitochondrial genome. Since 1988, mitochondrial DNA mutations have been linked to retinopathies, myopathies, neurodegenerative diseases, and possibly normal aging. Adequate drug therapies for these disorders have yet to be discovered. Therefore, gene therapy must be considered as a possible alternative. In this review, we will discuss the possibilities and the problems associated with gene therapy for mitochondrial disorders.
Owen
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Powell Gene Therapy Center, University of Florida Genetics Institute, Gainesville 32610-0266, USA.
Flotte
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TR
D000818:Animals; D001692:Biological Transport; D002467:Cell Nucleus / Q000235:genetics; D004272:DNA, Mitochondrial / Q000235:genetics; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D008928:Mitochondria / Q000235:genetics / Q000378:metabolism; D028361:Mitochondrial Diseases / Q000235:genetics / Q000628:therapy*; D024101:Mitochondrial Proteins / Q000235:genetics / Q000378:metabolism; D008954:Models, Biological; D009154:Mutation; D009696:Nucleic Acids / Q000378:metabolism; D015533:Transcriptional Activation
D004272:DNA, Mitochondrial; D024101:Mitochondrial Proteins; D009696:Nucleic Acids