基因治疗就是将遗传物质转入机体细胞达到治疗疾病的目的。这种技术不仅对遗传病有着广阔的应用前景,而且对于一些获得性疾病如癌症也可能有很大作用。目前基因治疗是通过体细胞研究来实现。基因治疗和器官,组织移植从根本上说是不同的,移植要解决供体组织和受体组织的排斥现象,而基因治疗则面临着基因高效转移和持久表达的挑战。在这篇综述中,我将总结在基因治疗遗传病方面取得的进展,着重讨论基因转移中受体靶组织。策略基因治疗的理想应用是将患病器官组织中有缺陷的基因进行精确修复,或将基因替 Gene therapy is the genetic material into the body cells to achieve the purpose of treatment of disease. This technique not only has broad application prospects for genetic diseases, but also may have a great effect on some acquired diseases such as cancer. At present gene therapy is achieved through somatic research. Gene therapy and organ and tissue transplantation are fundamentally different. Transplantation solves the exclusion of donor and recipient tissues, and gene therapy faces the challenge of efficient gene transfer and sustained expression. In this review, I will summarize the progress made in genetic therapy for gene therapy, focusing on receptor target tissues during gene transfer. The ideal application of strategy gene therapy is the precise repair of defective genes in diseased organs,