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

doi: 10.12290/xhyxzz.2023-0656

  • 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)

  • Received Date: 2023-12-27
  • Accepted Date: 2024-01-15
    • Available Online: 2024-03-05
    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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