Non-coding RNAs as drug targets

Masayuki Matsui, David R. Corey

Research output: Contribution to journalReview articlepeer-review

633 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)167-179
Number of pages13
JournalNature Reviews Drug Discovery
Issue number3
StatePublished - Mar 1 2017

ASJC Scopus subject areas

  • Pharmacology
  • Drug Discovery


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