基因治疗是一种新的治疗方法,即把一段外源基因导入患者的细胞,以纠正其先天性遗传缺陷或赋与患者的细胞以新的功能.本文将综述在发展一种实用而安全的基因转移方法方面已取得的进展,并讨论有关的基本原理和尚存在的困难和问题.一.总体上需加以考虑的问题体细胞基因治疗在基因治疗的最初阶段,将严格限于体细胞的基因治疗.即只把外源基因插入人体“患病”的细胞,而避免将其导入生殖细胞,否则就可能改变可遗传 Gene therapy is a new method of treatment that involves the introduction of a foreign gene into the patient’s cells to correct their congenital genetic defects or assign new functions to the patient’s cells. This article will review the development of a practical and safe Progress has been made in gene transfer methods, and related basic principles and difficulties are still discussed. I. General issues to be considered Somatic gene therapy will be strictly limited to the somatic cell gene therapy in the initial stage of gene therapy. That is, only the foreign gene is inserted into the “diseased” cell of the human body, and it is not possible to introduce it into the germ cell, otherwise it may change heritable