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

Jianle YANG; Nan WU

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

VernacularTitle:反义寡核苷酸的作用机制及其在运动系统中的研究进展
Author: Jianle YANG ¹ ; Nan WU ²
Author Information

1. Department of Orthopaedic Surgery, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China;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;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;
2. Department of Orthopaedic Surgery, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China;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;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;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;
Publication Type:Review
Keywords: antisense oligonucleotides; gene therapy; clinical practice; musculoskeletal system
From: Medical Journal of Peking Union Medical College Hospital 2024;15(4):891-896
CountryChina
Language:Chinese
Abstract:
Antisense oligonucleotides(ASOs) are a novel class of small molecule gene-targeted drugs that can bind with 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 have further advanced. This article reviews the mechanisms of ASOs in gene silencing and expression regulation, as well as their prospects in gene therapy. It also evaluates the research progress and applications of ASOs in musculoskeletal diseases and analyzes the urgent issues currently faced by this class of drugs. This comprehensive study aims to deepen our understanding of ASOs and provide valuable reference for their widespread application in biomedical research and clinical settings.