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抗心血管疾病的反义核酸药物研究进展

邵婉璇 黄渊余 樊渝川 殷涵 郭帅 李春辉 刘清 刘文静 唐晓英 翁郁华

生命科学仪器2023,Vol.21Issue(4):1-8,8.
生命科学仪器2023,Vol.21Issue(4):1-8,8.DOI:10.11967/2023210801

抗心血管疾病的反义核酸药物研究进展

Research Progresses of Antisense Oligonucleotides for Cardiovascular Diseases Therapy

邵婉璇 1黄渊余 1樊渝川 1殷涵 1郭帅 1李春辉 1刘清 1刘文静 1唐晓英 1翁郁华1

作者信息

  • 1. 北京理工大学生命学院,医学技术学院,前沿交叉科学研究院,分子医学与生物诊疗重点实验室,北京100081
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摘要

Abstract

With the understanding of the pathogenesis of diseases,nucleic acid drugs have become more and more impor-tant candidates for the treatment and intervention of diseases.In the traditional drug bank,only small parts of the targets associated with human disease have corresponding small molecule or protein drugs.In order to meet the extensive therapeu-tic needs of patients,it is urgent to develop drugs for the impossible targets of traditional drugs,and nucleic acid drugs can precisely target the previously impossible gene targets.The development of nucleic acid drugs that regulate gene expression,represented by antisense oligonucleotides,has accelerated in recent years.To date,there are many kinds of commercial an-tisense oligonucleotides,and many other drug candidates are under development.Although the vast majority of approved drug indications are for rare diseases,many antisense nucleic acid drugs currently in mid to late stage of clinical develop-ment are for common diseases,such as cardiovascular diseases.Thanks to advances in nucleic acid chemistry,the efficacy and safety of antisense oligonucleotide drugs have been significantly improved compared with decades ago.In this paper,the development history,mechanism and chemical modification techniques of antisense oligonucleotide drugs are reviewed,with emphasis on the development and research status of antisense oligonucleotide drugs in cardiovascular diseases,so as to provide theoretical basis and technical reference for the development of small nucleic acid drugs.

关键词

反义寡核苷酸/反义技术/心血管疾病/RNA剪接

Key words

Antisense oligonucleotide/Antisense Technology/Cardiovascular disease/RNA splicing

分类

医药卫生

引用本文复制引用

邵婉璇,黄渊余,樊渝川,殷涵,郭帅,李春辉,刘清,刘文静,唐晓英,翁郁华..抗心血管疾病的反义核酸药物研究进展[J].生命科学仪器,2023,21(4):1-8,8.

基金项目

国家自然科学基金(32001008,32171394,32101148,82202338). (32001008,32171394,32101148,82202338)

生命科学仪器

1671-7929

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