肿瘤基因治疗的研究现状和展望  

Tumor gene therapy: Current status and future prospectives

作　　者：邓洪新[1] 魏于全[1] 

机构地区：[1]四川大学华西医院生物治疗国家重点实验室,成都610041

出　　处：《中国肿瘤生物治疗杂志》2015年第2期170-176,共7页Chinese Journal of Cancer Biotherapy

基　　金：国家重点基础研究计划课题资助项目(No.2012CB917104);国家高技术研究发展计划(863计划)资助项目(No.2012AA020802);教育部新世纪优秀人才基金资助项目(No.NCET-11-0342)~~

摘　　要：基因治疗(gene therapy)是随着DNA重组技术、基因克隆技术等的成熟而发展起来的最具革命性的医疗技术之一,它是以改变人的遗传物质为基础的生物医学治疗手段。经过近三十年的发展,基因治疗已经由最初用于单基因遗传病的治疗扩大到恶性肿瘤、感染性疾病、心血管疾病、自身免疫性疾病、代谢性疾病等多种重大疾病的治疗,其中针对恶性肿瘤的基因治疗临床试验方案占了总数的2/3。本文将主要聚焦全球基因治疗的发展历史和我国肿瘤基因治疗的发展现状,重点介绍肿瘤基因治疗所用的表达载体、基因导入系统、临床试验、重点产品的研发和产业化发展以及近年来基因治疗在恶性肿瘤、重大遗传性疾病等治疗领域所取得的重点突破。此外,还从基因的体内递送、基因治疗的安全性、新技术用于肿瘤的基因治疗、肿瘤基因治疗与其他治疗方法的联合应用、基因检测技术与基因治疗相结合等五个方面,对未来肿瘤基因治疗所面临的发展机遇和挑战进行重点阐述。有理由相信,随着肿瘤基因治疗关键技术的不断突破,未来几年将是肿瘤基因治疗产品上市的重要时期,将为恶性肿瘤的临床治疗提供更多的新选择。Gene therapy is one of the most revolutionary medical technologies developing with the mature of the techniques of recombinant DNA and gene cloning and it is a kind of biomedical treatment based on changing human genetic materials. After nearly thirty years development,gene therapy has expanded from single-gene genetic diseases treatment to the therapy of malignant tumor,infectious diseases,cardiovascular disease,autoimmune disease,metabolic disease and other major diseases and about 2 /3 of the gene therapy clinical trials are directed to malignant tumor. This article will mainly focus on the development history of world gene therapy and the development status of tumor gene therapy in China with emphases on the reviews of gene expression vector,gene delivery system,gene therapy clinical trials,the research and development of gene therapy major products and the industrialization development of gene therapy and the recent key breakthroughs in the malignant tumor and genetic diseases gene therapy fields. Besides,this article reviews with emphasis on the development opportunities and challenges of tumor gene therapy in the future from five aspects including gene delivery in vivo,safety of gene therapy,new technology in tumor gene therapy,tumor gene therapy combined with other therapeutic methods and gene detection technology united with gene therapy. We have reasons to believe that along with the continuous breakthroughs of key technologies of gene therapy,the next few years will be an important period of tumor gene therapeutic products coming to the market,which provides new choices for the clinical trials of malignant tumor.

关 键 词：肿瘤 基因治疗 现状 展望 

分 类 号：R730.5[医药卫生—肿瘤]