靶向肝外器官的核酸药物研究进展及前景  

Progress and Prospect of Extrahepatic Targeting Nucleic Acid Drugs

作　　者：李硕蕾 杨雪华 毛楷凡 李大伟 刘正平 陈建英 张岱州 LI Shuo-lei;YANG Xue-hua;MAO Kai-fan;LI Da-wei;LIU Zheng-ping;CHEN Jian-ying;ZHANG Dai-zhou(Shandong Academy of Pharmaceutical Sciences,Jinan 250101,China;Shandong Engineering Research Center of New Sustained and Controlled Release Formulations and Drug Targeted Delivery Systems,Jinan 250101,China;Shandong Key Laboratory of Targeted Drug Delivery and Advanced Pharmaceutics,Jinan 250101,China)

机构地区：[1]山东省药学科学院,山东济南250101 [2]新型缓控释制剂与药物靶向递送系统山东省工程研究中心,山东济南250101 [3]山东省靶向递药与高端制剂重点实验室,山东济南250101

出　　处：《食品与药品》2024年第6期I0023-I0030,共8页Food and Drug

基　　金：山东省重点研发计划(重大科技创新工程)资助项目(编号:2021CXGC010515)。

摘　　要：作为一种新型药物治疗方案,核酸药物在肿瘤、遗传性疾病等的治疗中有良好的应用前景。现阶段核酸药物采用的药物递送系统靶向器官主要为肝脏,对于肝外组织和器官的特异性较差。这既是当前核酸药物研发和应用的优势,同时也是其局限性。本文对现有的靶向肝外器官的核酸药物的研究进展及前景进行综述,以为其研究和开发提供思路和帮助。As a new treatment modality,nucleic acid drugs have great potential in the treatment of human diseases such as cancer and genetic diseases.However,at the present stage,the drug delivery systems used by nucleic acid drugs are mainly liver-targeted and have poor specificity for extrahepatic tissues and organs.This is both a limitation and an opportunity for research and development of current nucleic acid drugs.This review mainly introduces the research progress and prospects of extrahepatic targeting nucleic acid drugs.

关 键 词：核酸药物 肝外疾病 递送系统 临床前研究 临床研究 

分 类 号：TQ464.6[化学工程—制药化工]