RNA药物递送载体的研究进展  

Research progress of vector for RNA drugs delivery

作　　者：娄瑞 林超龙 黄承浩[1] LOU Rui;LIN Chao-Long;HUANG Cheng-Hao(National Institute of Diagnostics and Vaccine Development in Infectious Diseases,Xiamen University,Xiamen 361102,China)

机构地区：[1]厦门大学国家传染病诊断试剂与疫苗工程技术究中心,厦门361102

出　　处：《生命科学》2023年第8期1012-1022,共11页Chinese Bulletin of Life Sciences

基　　金：国家自然科学基金项目(32100732)。

摘　　要：核糖核酸(ribonucleic acid,RNA)疗法通过将外源RNA引入特定细胞来精准调控基因的表达,从而实现对疾病的干预。近年来,临床结果表明RNA疗法在基因调控和疾病预防上是一种十分有潜力的基因疗法。然而,RNA药物在递送过程中仍然面临如稳定性差、组织靶向性弱、免疫原性强等诸多问题,限制了RNA药物的进一步临床转化应用。本文总结了不同RNA药物的递送方式在临床前和临床实验中取得的最新研究进展,并讨论不同递送载体的应用前景和局限性,旨在为RNA药物递送方式的优化提供新的设计思路,促进RNA疗法的临床应用。RNA-based therapies offer a promising approach for treating a variety of diseases by regulating gene expression through introduction of exogenous RNAs into target cells.Clinical data supports the potential of RNA therapeutics in gene regulation and disease prevention.However,RNA-based therapies face several challenges,such as poor stability,weak tissue targeting,and strong immunogenicity during delivery,which have hindered their clinical application.This review highlights recent progress in the development of various RNA drug delivery methods,both in preclinical and clinical trials,then discusses the potential and limitations of different delivery vectors,providing new insights for the optimization of RNA drug delivery methods and the advancement of clinical applications of RNA drugs.

关 键 词：核糖核酸 递送系统 非病毒载体 病毒载体 

分 类 号：R943[医药卫生—药剂学]