反义寡核苷酸药物在精准治疗中的应用进展及其作用机制  

APPLICATION AND MECHANISM OF ACTION OF ANTISENSE OLIGONUCLEOTIDES IN PRECISE TREATMENT

作　　者：周海燕 ZHOU Haiyan(University College London, London WC1N 1EH, United Kingdom)

机构地区：[1]伦敦大学学院,伦敦WC1N1EH

出　　处：《精准医学杂志》2020年第4期283-286,291,共5页Journal of Precision Medicine

摘　　要：反义寡核苷酸是一类新的基因靶向药物。随着基因组学与新一代基因测序技术的发展,以及各种新的核苷酸合成技术的出现,反义寡核苷酸药物在转化医学与精准医学中的应用取得了飞速的发展。该类药物为很多过去无法治疗的疾病提供了全新的治疗策略。本文以目前上市的几种代表性药物为例,集中阐释反义寡核苷酸药物在相关疾病治疗中的主要应用进展及作用机制,同时对该类药物开发中亟待解决的问题进行了分析。Antisense oligonucleotides(ASOs)are a new class of gene-targeted drugs,and with the development of genomics,next-generation sequencing techniques,and various new nucleotide synthesis techniques,ASOs have achieved rapid deve-lopment and wide application in translational medicine and precision medicine.ASOs provide new treatment strategies for diseases that cannot be treated in the past.With the examples of currently available ASO drugs on the market,this article summarizes the application and mechanism of action of ASOs in the treatment of related diseases and analyzes the problems that need to be solved in the development of such drugs.

关 键 词：寡核苷酸类 反义 遗传性疾病 先天性 基因治疗 靶基因修复 分子靶向治疗 精准医学 

分 类 号：R453[医药卫生—治疗学] R977[医药卫生—临床医学]