反义寡核苷酸治疗在神经退行性疾病中的应用  

Antisense oligonucleotide therapies in neurodegenerative disease

作　　者：王嘉(综述) 瞿宇晋(审校)[1] Wang Jia;Qu Yujin(Department of Medical Genetics,Capital Institute of Pediatrics,Beijing 100020,China)

机构地区：[1]首都儿科研究所遗传室,北京100020

出　　处：《国际儿科学杂志》2020年第5期297-301,共5页International Journal of Pediatrics

基　　金：北京市自然科学基金(7172039);首都儿科研究所方向性基金(方向-2017^02);国家重点研发项目(2016YFC0901505);中国医学科学院医学与健康科技创新工程(CAMS-I2M-1~008)。

摘　　要：反义寡核苷酸(antisense oligonucleotide,ASO)是一种新兴的治疗药物,通过序列特异地与疾病相关靶基因信使RNA结合,改变或减少靶基因的表达而发挥治疗作用。ASO药物具有特异性高、不良反应少等特点,被广泛应用于罕见病、肿瘤、心血管疾病、炎症类疾病、感染性疾病及代谢类疾病等的治疗领域。基于ASO的序列和化学修饰的改进,近年来在治疗神经退行性疾病中取得了突破性进展。Antisense oligonucleotides(ASOs)are small sequences of DNA or RNA able to target mRNA transcripts resulting in change or reduction in the expression of target genes,and became an emerging class of therapeutic drugs.With its high specific,adaptable,and low side effects,ASO drugs are widely used in rare diseases,tumor,inflammatory diseases,cardiovascular disease,and infectious diseases.Recent numerous advancements in ASO sequence design,chemical modifications and delivery methods,significant breakthroughs have been made in the treatment and ASOs become an ideal candidate therapies for neurodegenerative diseases.

关 键 词：反义寡核苷酸 神经退行性疾病 化学修饰 治疗 

分 类 号：R73[医药卫生—肿瘤]