小核酸药物的研究进展

Current advances of small nucleic acid therapeutics

小核酸药物作用于细胞内致病靶基因和RNA片段,通过改变宿主遗传信息的方式和调控蛋白表达达到治疗疾病的目的。其主要包括小干扰RNA(small interfering RNA,siRNA)、微核苷酸(microRNA,miRNA)和反义寡核苷酸(antisense oligonucleotides,ASO)。小核酸药物在临床上的应用较为广泛,包括罕见病、慢性病和肿瘤等。现就小核酸药物的主要分类、作用机制和临床应用等作一阐述。

Small nucleic acid drugs,mainly including small interfering RNA(siRNA),microRNA(miRNA),and antisense oligonucleotides(ASO),primarily treat diseases by targeting on intracellular pathogenic genes and RNA fragments to alter host cell genetic informations and regulate protein expressions.These drugs are widely used as therapeutic macromolecules for the treatment and prevention of diverse diseases,ranging from rare disorders,chronic diseases to various types of tumors.This article reviews the main classification,the related mechanism of action and clinical application of small nucleic acid drugs.