Non-coding RNAs as drug targets

Abstract

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.