细胞外囊泡介导的小干扰RNA药物递送系统研究进展  

Advances in Extracellular Vesicle-Mediated siRNA Delivery System

作　　者：李睿 朱梦玺 胡茜雯 孙璐 贺慧宁 LI Rui;ZHU Mengxi;HU Xiwen;SUN Lu;HE Huining(Tianjin Municipal Key Laboratory of Clinical Pharmaceutical Technology,School of Biomedical Engineering and Technology,School of Pharmacy,Tianjin Medical University,Tianjin 300070,China)

机构地区：[1]天津医科大学药学院生物医学工程与技术学院,天津市临床药物关键技术重点实验室,天津300070

出　　处：《药学进展》2025年第1期15-23,共9页Progress in Pharmaceutical Sciences

基　　金：国家自然科学基金项目(No.82173769)。

摘　　要：小干扰RNA(small interfering RNA,siRNA)因其具有基因沉默效率高、特异性强、靶点范围广等优势,成为极具潜力的治疗药物,然而因其难以自主入胞、靶向性差等问题,临床应用也面临诸多挑战。细胞外囊泡作为一种天然纳米级膜囊泡,被证明是siRNA药物递送的优良载体,可保护siRNA免受核酸酶降解,并增强细胞摄取和胞内转运能力,在提高siRNA生物活性和细胞/器官选择性方面具有独特的优势。通过对细胞外囊泡介导的siRNA递送策略进行综述,并讨论影响其临床应用的主要因素和该领域的未来发展方向,以期为siRNA药物的递送和开发提供参考。Small interfering RNA(siRNA)presents great potential in therapeutic application with distinct advantages including high efficiency of gene silencing,strong specificity,and wide range of targeting,however,its clinical application also faces challenges due to its poor cell membrane permeability and targeting efficiency.Extracellular vesicles,a kind of natural nanovesicles,have been proved to be excellent carriers for siRNA drug delivery.They can be used to protect siRNA from nuclease degradation,and enhance cellular uptake and cytoplasmic transport,demonstrating unique advantages in enhancing the biological activity and cell/organ selectivity of siRNA.This review highlights the strategies for extracellular vesicle-mediated siRNA delivery,and further discusses major considerations and future directions in this field,in order to provide some reference for the delivery and development of siRNA therapeutics.

关 键 词：小干扰RNA 细胞外囊泡 药物递送系统 

分 类 号：R94[医药卫生—药剂学]