基因治疗(Gene therapy)是指通过遗传操作将具有正常功能的基因导入患者细胞中,替代或补足失去的功能,以达到在基因水平上治疗遗传病的目的。目前已知的小儿遗传病有3000种以上,大多数为单基因遗传病。对于遗传病,既往使用传统治疗方法,如苯酮尿症、半乳糖血症的饮食疗法,地中海贫血、血友病的反复输血治疗等,这些治疗既不能达到根治的目的,又有可能造成免疫反应和病毒感染等 Gene therapy refers to the genetically-engineered gene into the patient’s cells to replace or complement the lost function, in order to achieve the genetic level of genetic disease treatment purposes. At present, there are more than 3000 pediatric genetic diseases known, most of them are single-gene genetic diseases. For genetic diseases, the traditional treatment of past use, such as phenylketonuria, galactosemia diet, thalassemia, hemophilia repeated blood transfusion therapy, these treatments can not achieve the purpose of radical, but also may cause immune response And virus infection