基因治疗是遗传工程在医学治疗方面的应用。该方法是将外源性目的基因导入患者的细胞后,再输回患者,通过细胞表达转移的正常基因使疾病得到治疗。基因治疗的疾病主要包括遗传性疾病、免疫缺损和肿瘤等等。目前,基因治疗已有成功的报道,但还存在一些问题。外源性目的基因的制备,基因导入靶细胞的方式,靶细胞的类型以及基因表达的调控等是决定治疗成败的关键。 Gene therapy is the application of genetic engineering in medical treatment. In this method, the exogenous gene of interest is introduced into the patient’s cell and then returned to the patient, and the disease is treated by expressing the normal gene transferred by the cell. Gene therapy diseases include genetic diseases, immune defects and tumors and so on. At present, gene therapy has been successfully reported, but there are still some problems. Preparation of exogenous target gene, gene into target cells, the type of target cells and gene expression regulation is the key to the success or failure of treatment.