Non-coding RNAs as drug targets

被引：855

作者：

Matsui, Masayuki ^{[1
,2
]}

Corey, David R. ^{[1
,2
]}

机构：

[1] UT Southwestern, Dept Pharmacol, Dallas, TX 75390 USA

[2] UT Southwestern, Dept Biochem, Dallas, TX 75390 USA

来源：

NATURE REVIEWS DRUG DISCOVERY | 2017年 / 16卷 / 03期

基金：

美国国家卫生研究院;

关键词：

ANTISENSE OLIGONUCLEOTIDES; BCL-2; ANTISENSE; MOLECULAR-MECHANISMS; BINDING-SPECIFICITY; MYOTONIC-DYSTROPHY; CRITICAL REGULATOR; ADVANCED MELANOMA; ANGELMAN SYNDROME; MESSENGER-RNA; NUCLEAR-RNA;

D O I：

10.1038/nrd.2016.117

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

Most of the human genome encodes RNAs that do not code for proteins. These non-codingRNAs (ncRNAs) may affect normal gene expression and disease progression, making them a new class of targets for drug discovery. Because their mechanisms of action are often novel, developing drugs to target ncRNAs will involve equally novel challenges. However, many potential problems may already have been solved during the development of technologies to target mRNA. Here, we discuss the growing field of ncRNA including microRNA, intronic RNA, repetitive RNA and long non-coding RNA and assess the potential and challenges in their therapeutic exploitation.

引用

页码：167 / 179

页数：13

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