Approaches and limitations to gene therapy for mitochondrial diseases

Abstract

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.