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Journal Article
Non-coding RNAs as drug targets
2017
Overview
Key Points
Non-coding RNAs (ncRNAs) — which include microRNAs (miRNAs), repetitive RNAs, intronic RNAs, and long ncRNAs (lncRNAs) — are a diverse group of biomolecules with broad potential to control gene expression.
Compounds that target ncRNAs have the potential to control expression of disease-related genes
Development of compounds to target ncRNAs can benefit from understanding the lessons learnt from decades of research using antisense oligonucleotides (ASOs) and duplex RNAs to control expression of mRNA.
ASOs that affect splicing or that are complementary to miRNAs are already being tested in multiple clinical trials in a variety of diseases, including cancer and muscular dystrophy
lncRNAs can affect transcription or splicing and are emerging as a promising class of novel drug targets.
Non-coding RNAs (ncRNAs) may affect normal gene expression and disease progression, thereby representing potential drug targets. Here, Matsui and Corey assess the potential and challenges in therapeutically exploiting ncRNA species — including microRNA, intronic RNA, repetitive RNA and long ncRNA — highlighting key lessons learned during the development of technologies targeting mRNA.
Most of the human genome encodes RNAs that do not code for proteins. These non-coding RNAs (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.
Publisher
Nature Publishing Group UK,Nature Publishing Group
