siRNA新型非病毒载体递送策略新进展  

Recent Advances in Novel Non-viral Vector Delivery Strategies for siRNA

作　　者：陶钰萍 赵珍玉 李又欣[1] 孙考祥[1] 史亚楠 TAO Yuping;ZHAO Zhenyu;LI Youxin;SUN Kaoxiang;SHI Yanan(Key Lab.of Molecular Pharmacology and Drug Evaluation,Ministry of Education,Collaborative Innovation Center of Advanced Drug Delivery System and Biotech Drugs in Universities of Shandong,School of Pharmacy,Yantai University,Yantai 264005;College of Life Science,Yantai University,Yantai 264005)

机构地区：[1]烟台大学药学院,山东省高校新型制剂与生物技术药物研究协同创新中心,分子药理和药物评价教育部重点实验室,山东烟台264005 [2]烟台大学生命科学学院,山东烟台264005

出　　处：《中国医药工业杂志》2023年第4期471-480,共10页Chinese Journal of Pharmaceuticals

基　　金：山东省自然科学基金面上项目(ZR2021MH395)。

摘　　要：小干扰RNA(siRNA)是一个靶向治疗和精确医学的代表性治疗工具,可通过序列特异性的RNA干扰(RNAi)沉默任何疾病相关基因的表达。然而,它的治疗前景历来受到体内半衰期短、递送困难和安全问题的限制。非病毒载体介导的药物递送已经成为克服这些局限性的一个成功策略,可实现siRNA在体内的有效递送,高效沉默靶基因。目前,已有多种药物处于临床试验中,4种基于siRNA的新型疗法已获得美国FDA的批准,标志着靶向疗法新时代的开始。该文概述了近年来基于siRNA的非病毒载体递送策略的新进展及其应用,并展望了siRNA药物研究的未来发展趋势。Small interfering RNA(siRNA),a representative therapeutic tool for targeted therapy and precision medicine,can silence the expression of any disease-related gene through sequence-specific RNA interference(RNAi).However,its therapeutic prospects have historically been limited by short half-lives in vivo,difficulties in delivery,and safety concerns.Non-viral vector drug delivery has efficiently achieved siRNA delivery in vivo and silenced the target gene,becoming a successful strategy to overcome above limitations.Currently,several drugs are already in clinical trials,and four new siRNA-based therapies have been approved recently by the U.S.FDA,marking the beginning of a new era of targeted therapies.This review summarizes the recent development and application of siRNA-based non-viral vector delivery strategies and predicts the future development trend of siRNA drug research.

关 键 词：RNA干扰 siRNA药物 基因沉默 药物递送 基因治疗 

分 类 号：R944.9[医药卫生—药剂学]