siRNA注射给药系统研究进展  

Advances in Injectable siRNA Delivery Systems

作　　者：刘宵钰 闫仪 王坚成[1] LIU Xiaoyu;YAN Yi;WANG Jiancheng(Dept,of Pharmaceutics,School of Pharmaceutical Sciences,Peking University,Beijing 100191)

机构地区：[1]北京大学药学院药剂学系

出　　处：《中国医药工业杂志》2019年第10期1113-1125,共13页Chinese Journal of Pharmaceuticals

基　　金：国家自然科学基金(81773650、81973259);国家新药创制重大专项(2018ZX09721003-004)

摘　　要：siRNA作为RNA干扰技术的重要效应分子,可以特异性沉默靶基因,在疾病治疗领域具有较好的应用潜力。然而,作为一种负电性核酸分子,siRNA在临床疾病治疗应用中仍面临诸多挑战,如被RNA酶降解、体内快速清除、跨膜转运能力差、生物利用率低等问题。因此,安全高效的递送载体的研究开发是siRNA成药的关键。目前已有诸多将生物相容性纳米载体应用于siRNA体内注射给药的研究,并在临床或临床前研究中取得了较好的应用效果。本文详细综述了siRNA注射给药系统的结构和功能特征,整理分析了现有siRNA注射给药系统的优势和不足,探讨了siRNA药物研究的未来发展趋势。As an important effector of RNA interference,siRNA can specifically silence target genes,so it has a good potential for application in the field of disease treatment.However,as a negatively charged macromolecular nucleic acid,siRNA still faces many challenges in the treatment of clinical diseases,such as RNase degradation,rapid clearance in vivo,poor transmembrane capacity,and low bioavailability.Therefore,it is important to develop safe and efficient delivery systems for siRNA.At present,there are many studies on injection of biocompatible nanocarriers for siRNA in vivo,which have achieved great outcomes in clinical or preclinical trials.This article will review the structure and function of injectable siRNA delivery systems,analyze their advantages and disadvantages,and explore the development trend of siRNA delivery in the future.

关 键 词：SIRNA 注射给药 载体系统 基因沉默 

分 类 号：R944.9[医药卫生—药剂学]