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基因治疗最主要的对象之一是恶性肿瘤,因为肿瘤常规治疗的方法如手术、放疗及化疗等由于缺乏特异性和对正常组织毒性太大而受到限制。基因治疗针对肿瘤发生的遗传基础,容许选择性地破坏肿瘤细胞,同时减少系统毒性;而且肿瘤基因治疗基本上不用考虑社会伦理问题,可选用的治疗基因较多,实验模型易建立,患者及家属易接受及临床治疗也迫切需要。因此,尽管基因治疗是由遗传病开始,但肿瘤基因治疗后来居上,发展迅速。自1989年至1996年美国食品及药品管理局(FDA)批准基因治疗进入临床试验的治疗方案已有120多项,而肿瘤基因治疗方案占80％以上。
One of the most important targets of gene therapy is malignancy because conventional methods of tumor treatment such as surgery, radiotherapy, and chemotherapy are limited due to lack of specificity and toxicity to normal tissue. Gene therapy is aimed at the genetic basis of tumorigenesis, allowing selective destruction of tumor cells, while reducing systemic toxicity; and tumor gene therapy basically does not have to consider social ethical issues, the available therapeutic genes are more, experimental models are easy to establish, patients and their families Easy acceptance and clinical treatment are also urgently needed. Therefore, although gene therapy started with genetic diseases, tumor gene therapy came first and it developed rapidly. From 1989 to 1996, the United States Food and Drug Administration (FDA) approved more than 120 treatment programs for gene therapy into clinical trials, and tumor gene therapy programs accounted for more than 80%.