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反义寡核苷酸的作用机制及其在运动系统中的研究进展

杨建乐,  吴南

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文章导航 > 协和医学杂志  > 2024 > 优先出版
杨建乐, 吴南. 反义寡核苷酸的作用机制及其在运动系统中的研究进展[J]. 协和医学杂志. doi: 10.12290/xhyxzz.2023-0656
引用本文: 杨建乐, 吴南. 反义寡核苷酸的作用机制及其在运动系统中的研究进展[J]. 协和医学杂志. doi: 10.12290/xhyxzz.2023-0656
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YANG Jianle, WU Nan. Mechanism of Action of Antisense Oligonucleotides and Their Research Progress in the Musculoskeletal System[J]. Medical Journal of Peking Union Medical College Hospital. doi: 10.12290/xhyxzz.2023-0656
Citation: YANG Jianle, WU Nan. Mechanism of Action of Antisense Oligonucleotides and Their Research Progress in the Musculoskeletal System[J]. Medical Journal of Peking Union Medical College Hospital. doi: 10.12290/xhyxzz.2023-0656
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反义寡核苷酸的作用机制及其在运动系统中的研究进展

doi: 10.12290/xhyxzz.2023-0656

  • 1 中国医学科学院北京协和医院 骨科, 北京 100730;

  • 2 中国医学科学院北京协和医院 骨骼畸形遗传学研究北京市重点实验室, 北京 100730;

  • 3 中国医学科学院北京协和医院 脊柱畸形大数据研究与应用重点实验室, 北京 100730;

  • 4 中国医学科学院北京协和医院 疑难重症及罕见病国家重点实验室, 北京 100730

基金项目: 

中国医学科学院医学与健康科技创新工程（2021-I2M-1-051）； 北京协和医院中央高水平医院临床科研专项（2022-PUMCH-C-033）

详细信息
    通讯作者:

    吴南,E-mail:dr.wunan@pumch.cn

  • 中图分类号: R681； R746

Mechanism of Action of Antisense Oligonucleotides and Their Research Progress in the Musculoskeletal System

  • 1 Department of Orthopaedic Surgery, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China;

  • 2 Beijing Key Laboratory for Genetic Research of Skeletal Deformity, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China;

  • 3 Key Laboratory of Big Data for Spinal Deformities, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China;

  • 4 State Key Laboratory of Complex Severe and Rare Diseases, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China

Funds: 

CAMS Innovation Fund for Medical Sciences (2021-I2M-1-051)； National High Level Hospital Clinical Research Funding (2022-PUMCH-C-033)

    摘要
  • 摘要: 反义寡核苷酸（antisense oligonucleotides，ASOs）是一类新型的小分子基因靶向药物，可与目的mRNA结合，ASOs以其与目标序列互补的碱基配对方式，对基因进行靶向调控。随着基因测序技术和分子合成技术的不断发展，ASOs在运动系统中的研究和应用进一步深入。本文阐述了ASOs在基因沉默和表达调控中的作用机制，以及其在基因治疗方面的应用前景。此外，还介绍了ASOs在运动系统疾病中的研究进展和应用情况，并分析此类药物目前所面临的亟待解决的问题，旨在深化医务人员对ASOs的认识，并为其在生物医学研究和临床中的推广应用提供有益参考。
    Abstract: Antisense oligonucleotides (ASOs) are a novel class of small molecule gene-targeted drugs that can bind to target mRNA. Through complementary base pairing with the target sequence， antisense oligonucleotides achieve targeted regulation of genes. With the continuous development of gene sequencing technology and molecular synthesis techniques， research and applications of ASOs in the musculoskeletal system are further advancing. This article reviews the mechanisms of ASOs in gene silencing and expression regulation， as well as their prospects in gene therapy. In addition， we evaluate the research progress and applications of ASOs in musculoskeletal diseases， while analyzing the urgent issues currently faced by this class of drugs. This comprehensive study aims to deepen our understanding of ASOs and provide valuable references for their widespread application in biomedical research and clinical settings.
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    • 参考文献(30)
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出版历程
  • 收稿日期:  2023-12-27
  • 录用日期:  2024-01-15
  • 网络出版日期:  2024-03-05

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