线粒体病的基因治疗

线粒体病是一组因线粒体DNA缺失或突变而致氧化磷酸化及能量供应异常的疾病,目前该类疾病的治疗主要是支持治疗。然而由于线粒体结构和功能的特殊性,该疗效并不确切。因此,线粒体病的基因治疗显得越来越迫切和重要。目前,基因治疗的策略包括降低突变型mtDNA/野生型mtDNA的比例、错位表达、输入其他同源性基因以及利用限制性内切酶修复突变型mtDNA等。

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.