基因治疗药物输递系统的研究现状及发展趋势  

The Advance of Gene Delivery Vectors

作　　者：李燕[1,2] 阳俊[1] 刘桂英[3] 张欣[1] 

机构地区：[1]中国科学院过程工程研究所,生化工程国家重点实验室,北京100190 [2]中国科学院大学,北京100049 [3]首都医科大学附属北京安贞医院儿科,北京100029

出　　处：《生物化学与生物物理进展》2013年第10期998-1007,共10页Progress In Biochemistry and Biophysics

基　　金：国家自然科学基金(51103159;51203162);国家高技术研究发展计划(863)(2012AA022703;2012AA020804)资助项目~~

摘　　要：基因治疗是一种有效的治疗方法,可用于治疗多种严重威胁人类健康的疾病.然而,裸露的基因治疗药物存在易被核酶降解、细胞内吞效果差和细胞靶向能力差等缺点.因此,需要寻求合适的载体,将基因治疗药物有效地输递到靶细胞,实现高效的基因治疗.本文主要综述了近年来基因治疗药物输递系统的研究进展,分别总结和阐述了病毒载体,脂质体、聚合物和树状大分子等非病毒载体,以及具有示踪功能的输递系统的特点及研究和发展现状.Gene therapy has rapidly emerged as a promising method for the treatment of numerous diseases. However, the intrinsic deficiencies of gene-based drugs, such as enzymatic degradation in the body, poor membrane penetrability and lack of target ability, have limited the development of its therapeutic application. Herein, it is important for efficient gene therapy to develop efficient and safe gene vectors that can deliver the gene-based drugs to targeted cells. This paper reviews the current development in gene delivery vectors, especially summarizes the characteristic and development of viral vectors, non-viral vectors and nanoparticles with tracing ability.

关 键 词：基因治疗 病毒载体 脂质体 聚合物 树状大分子 

分 类 号：R452[医药卫生—治疗学] Q7[医药卫生—临床医学]