基因治疗是通过对一个需要长期表达基因的设计,将其植回宿主细胞并成功地得到表达,以替代缺陷基因或特异地抑制某些致病介质表达,从而达到治疗目的的方法。有人将其称为分子治疗(moleculartherapy)。基因治疗的问世是疾病治疗学的一场革命,也为人类征服疾病开拓了一条充满希望 Gene therapy is achieved through the design of a gene that requires long-term expression, which is then replanted into host cells and successfully expressed to replace defective genes or to specifically inhibit the expression of certain pathogenic agents to achieve therapeutic goals. Some people call it molecular treatment (moleculartherapy). The advent of gene therapy is a revolution in the treatment of diseases and opens up a promising future for human conquest of diseases