RNA药物非病毒递送系统研究进展  

Advances in Research on Non-viral Delivery Systems for RNA Drugs

作　　者：陈欢 魏利军 CHEN Huan;WEI Lijun(Product Portfolio Department,Harbin Pharmaceutical Group Co.,Ltd.,Beijing 100023,China)

机构地区：[1]哈药集团股份有限公司产品立项部,北京100023

出　　处：《药学进展》2022年第11期839-847,共9页Progress in Pharmaceutical Sciences

摘　　要：RNA疗法通过将外源性的RNA引入特定细胞来调控基因表达,是一种非常有潜力的疾病治疗策略。然而,由于存在体内稳定性差、难以高效进入靶细胞等问题,RNA药物的递送需借助合适的药物递送系统。与病毒载体相比,非病毒载体具有更高的安全性,已成为本领域的研究热点。主要介绍脂质纳米颗粒递送系统、生物偶联递送系统以及外泌体递送系统这3种可帮助RNA疗法进入临床的非病毒载体递送系统,并对其技术进展和难点进行分析与总结。RNA therapy, which regulates gene expression by introducing exogenous RNAs into specific cells, is a very promising strategy for disease treatment. However, the delivery of RNA drugs require suitable drug delivery systems, because RNAs are unstable in vivo and cannot enter target cells efficiently. Compared with viral vectors, non-viral vectors are safer, and have become a research hotspot. In this article, we mainly introduced three non-viral delivery systems that may enable the clinical translation of RNA therapeutics:lipid nanoparticle delivery systems, bioconjugation delivery systems, and exosome delivery systems. The related technical progress and difficulties were also analyzed and summarized.

关 键 词：RNA药物 非病毒递送系统 脂质纳米粒 生物偶联 外泌体 

分 类 号：R945[医药卫生—微生物与生化药学]