Gene Therapy of Mitochondrial Diseases

The mitochondrial diseases derived from defects in mitochondrial DNA(mtDNA)encompass a diverse group of disorders resulting in oxidative phosphorylation dysfunction and energy supply deficiency.Currently,treatment of the diseases remains supportive therapy,but the results are far from satisfactory due to the special structure of mitochondria.As a consequence,gene therapy is becoming increasingly important for the treatment of the diseases.And now the strategies of gene therapy include decreasing the ratio of mutant to wild-type mitochondrial genomes,allotropic expression,importing cognate genes from other species,or correcting mtDNA mutations with specific restriction endonucleases.