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 with target mRNA.Through complementary base pairing with the target sequence,antisense oligo-nucleotides 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 wide-spread application in biomedical research and clinical settings.
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)
中央高水平医院临床科研专项(2022-PUMCH-C-033)
国家科技期刊平台
NSTL
万方数据