中药及天然药物来源的小核酸新药发现与前景展望  

New drug discovery and prospect of small nucleic acids derived from traditional Chinese medicine and natural medicine

作　　者：华桦 田韦韦 刘俐 曾瑾 魏平 谭蕊蓉 王剑波 赵军宁 HUA Hua;TIAN Wei-wei;LIU Li;ZENG Jin;WEI Ping;TAN Rui-rong;WANG Jian-bo;ZHAO Jun-ning(Sichuan Institute for Translational Chinese Medicine,Key Lab of Biological Evaluation of TCM Quality of the State Administration of Traditional Chinese Medicine,Translational Chinese Medicine Key Laboratory of Sichuan Province,Sichuan Engineering Technology Research Center of Genuine Regional Drug,Sichuan Provincial Engineering Research Center of Formation Principle and Quality Evaluation of Genuine Medicinal Materials,Chengdu 610041,China;National Key Laboratory of Drug Regulatory Science,National Medical Products Administration,Beijing 100037,China)

机构地区：[1]四川省中医药转化医学中心,国家中医药管理局中药质量生物评价重点研究室,中医药转化医学四川省重点实验室,四川省道地药材系统开发工程技术研究中心,四川省道地药材形成原理与品质评价工程研究中心,四川成都610041 [2]国家药品监督管理局药品监管科学全国重点实验室,北京100037

出　　处：《中国中药杂志》2024年第9期2273-2280,共8页China Journal of Chinese Materia Medica

摘　　要：小核酸药物主要包括小干扰核糖核酸(small interfering RNA,siRNA)、反义寡核苷酸(antisense oligonucleotide,ASO)、微小RNA(microRNA,miRNA)、信使RNA(messenger RNA,mRNA)以及核酸适配体(aptamer)等,可通过与靶分子的RNA结合,抑制其翻译或调控,具有高度特异性、持久性和可治愈性,近年来颇受关注。新近发现中药动植物来源的miRNA自身稳定并可稳定存在于动物或人体的血液与组织器官,而且可通过参与调控多种靶蛋白的表达发挥药理作用。该文概述了源自中药及天然药物的小核酸药物发现及其跨界调控机制,探讨这类新兴药品带来的技术挑战与监管问题,可为中药复杂性作用机制阐释、中药及天然药物来源的小核酸新药研制及监管科学研究提供新的思路和方法。Small nucleic acid drugs mainly include small interfering RNA(siRNA),antisense oligonucleotide(ASO),microRNA(miRNA),messenger RNA(mRNA),nucleic acid aptamer(aptamer),and so on.Its translation or regulation can be inhibited by binding to the RNA of the target molecule.Due to its strong specificity,persistence,and curability,small nucleic acid drugs have re-ceived considerable attention in recent years.Recent studies have shown that some miRNAs from animal and plant sources can stably exist in the blood,tissue,and organs of animals and human beings and exert pharmacological action by regulating the expression of va-rious target proteins.This paper summarized the discovery of small nucleic acids derived from traditional Chinese medicine(TCM)and natural drugs and their cross-border regulatory mechanisms and discussed the technical challenges and regulatory issues brought by this new drug,which can provide new ideas and methods for explaining the complex mechanism of TCM,developing new drugs of small nu-cleic acids from TCM and natural medicine,and conducting regulatory scientific research.

关 键 词：小核酸 中药 天然药物 新药发现 监管科学 

分 类 号：R285[医药卫生—中药学]