Portal de Periódicos da CAPES

Non-coding RNAs as drug targets

2016; Nature Portfolio; Volume: 16; Issue: 3 Linguagem: Inglês

10.1038/nrd.2016.117

1474-1784

Masayuki Matsui, David R. Corey,

MicroRNA in disease regulation

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.