Development of in vivo mitochondrial gene delivery system and validation of mitochondrial exogenous gene expression in skeletal muscle

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Mitochondrial genetic disorders are a major cause of mitochondrial diseases including mitochondrial inherited diseases, neurodegenerative diseases and diabetes. It is therefore likely that mitochondrial gene therapy will be useful for the treatment of such diseases. To achieve such an innovative therapy, successful mitochondrial exogenous gene expression, two independent processes, i.e., "development of in vivo mitochondrial gene delivery system" and "construction of DNA vector to achieve mitochondrial gene expression" are required. In this study, we report on successful mitochondrial gene delivery in skeletal muscle using hydrodynamic limb vein (HLV) injection. Moreover, we could construct the DNA vector which can express on mitochondria through hydrodynamic gene delivery. Our mitochondrial exogenous gene expression system promises to be a useful technique for mitochondrial gene therapy and research regarding mitochondrial molecular biology.