3. Gene therapy: Confronting mitoch

3. Gene therapy: Confronting mitochondrial pathologiesAlthough contemporary research has considerably enhanced our understanding of the geneticand biochemical etiology of mitochondrial disorders, therapeutic interventions for the samehave been preliminary at best. Nevertheless, with recent advances in technology, gene therapyhas emerged as a potential strategy to address mitochondrial diseases, either by manipulatingthe nuclear genome or by targeting the mitochondrial paraphernalia (Weissig and Torchilin,2001). Gene therapy is the replacement of a defective gene by delivering a corrective alleleinto the host cell, or silencing a dominant mutant allele that is pathogenic to the host (Kaufmann