siRNA药物研究进展  

Research Progress of siRNA Drugs

作　　者：贺婉红[1] 薛嫚[1] 李芳[1] 俸灵林[1] 张婷[1] HE Wan-hong;XUE Man;LI Fang;FENG Ling-lin;ZHANG Ting(National Population and Family Planning Key Labo- ratory of Contraceptives Drugs and Devices,Shanghai Engineering Research Center of Reproductive Health Drug and Devices,Shanghai Institute of Planned Parenthood Research,Shanghai 200032,Chin)

机构地区：[1]国家人口和计划生育委员会计划生育药具重点实验室上海生殖健康药具工程技术研究中心上海市计划生育科学研究所,上海200032

出　　处：《中国药学杂志》2018年第14期1145-1151,共7页Chinese Pharmaceutical Journal

基　　金：上海市2014年度"科技创新行动计划"生物医药领域科技支撑项目资助(14431902000);上海市科学技术委员会科研计划项目资助(14JC1492300)

摘　　要：RNA干扰技术(RNAi)因其具有高效、高特异性、低毒等优点在生物医药领域方面潜力巨大,为新药的研发提供了非常好的思路。通过合理设计、化学修饰及载体可在一定程度上解决小干扰RNA(siRNA)药物快速降解、细胞摄取率低和脱靶效应等问题。笔者主要从适应证、给药方式及纳米载体等几个方面对进入临床试验的siRNA药物进行综述,为从事siRNA药物研发的研究者提供参考。RNA interference technology（RNAi） has a great potential in the biomedical field, providing a very good idea for the re- search and development of new drugs because of its high efficiency, high specificity, and low toxicity. Rational design strategies, chemi- cal modifications and nanocarriers offer significant opportunities to overcome the deficiencies of siRNA drugs such as rapid degradation, poor cell uptake and off-target effects. Here, the research progress of some siRNA drugs which have entered clinical trials was reviewed based on their indications, administration routes and nanocarriers, which provides references for researchers in development of siRNA drugs.

关 键 词：小干扰RNA 基因沉默 治疗应用 纳米载体 

分 类 号：R944[医药卫生—药剂学]