摘要
小干扰RNA(siRNA)是一种新型核酸药物,其通过RNA干扰(RNAi)机制,在转录后水平靶向沉默靶基因的表达,从而发挥治疗疾病的作用。siRNA作为一种精准、特异、高效的基因沉默疗法,在肿瘤靶向治疗领域备受瞩目。目前,已有多款抗肿瘤siRNA疗法进入临床试验阶段。综述了近年来siRNA在肿瘤靶向治疗中的研究进展,包括siRNA作用机制、抗肿瘤siRNA药物研发近况,以及siRNA药物体内递送的优化策略,旨在为抗肿瘤siRNA药物的研发与临床应用提供新思路。
Small interfering RNA(siRNA)is a new type of nucleic acid drug that plays its therapeutic roles through the RNA interference(RNAi)mechanism to target and silence the expression of target genes at the post-transcriptional level.As a precise,specific and efficient gene silencing therapy,siRNA has attracted much attention in the field of tumor-targeted therapy.Currently,several anti-tumor siRNA therapies have entered the clinical trial stage.This article summarizes the research progress of siRNA in tumor-targeted therapy in recent years,including the mechanism of action for siRNA,the recent development of anti-tumor siRNA drugs,and the optimization strategies for the in vivo delivery of siRNA drugs,aiming to provide new insights for the development and clinical application of anti-tumor siRNA drugs.
作者
张从一
牧原
李振海
王欣月
蔡文秋
刘晓婧
邹倩
李梦玮
ZHANG Congyi;MU Yuan;LI Zhenhai;WANG Xinyue;CAI Wenqiu;LIU Xiaojing;ZOU Qian;LI Mengwei(School of Life Science and Technology,China Pharmaceutical University,Nanjing 210009,China)
出处
《药学进展》
CAS
2024年第7期548-560,共13页
Progress in Pharmaceutical Sciences
基金
国家自然科学基金(No.82103118)
江苏省自然科学基金(No.BK20210418)。
关键词
RNA干扰
小干扰RNA
肿瘤靶向治疗
肝外靶向
纳米载体
RNA interference
small interfering RNA
tumor-targeted therapy
extra-hepatic targeting
nanocarrier
