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对遗传病进行体细胞基因治疗,其方法与自体组织移植相似.用反转录病毒载体通过包装细胞系转染离体组织细胞再植入患者体内,转移基因在体内适宜表达即可纠正遗传病.其焦点是转移系统的安全性与有效性和转移基因在受体细胞内表达的适宜性.用反转录病毒载体已成功地将多种基因转移至造血细胞、皮肤细胞、肝细胞等组织细胞,在这些细胞和整体动物内得到表达。临床已试验治疗先天性免疫缺陷综合症并初见成效,说明对遗传病进行体细胞基因治疗是可行的。
Somatic gene therapy for hereditary diseases is similar to that of autologous tissue transplantation.Retroviral vectors are used to transfect ex vivo tissue cells through packaging cell lines and then re-implanted into the body of patients for gene therapy The focus is on the safety and efficacy of the metastatic system and the suitability of the transgene to be expressed in recipient cells.Retroviral vectors have been used to successfully transfer multiple genes to tissues such as hematopoietic cells, skin cells, and hepatocytes Cells are expressed in these cells and in whole animals. Clinical trials have proven effective in treating congenital immunodeficiency syndrome, indicating that it is feasible to carry out somatic gene therapy for genetic diseases.