Synthetic RNA Modulators in Drug Discovery

J Med Chem. 2021 Jun 10;64(11):7110-7155. doi: 10.1021/acs.jmedchem.1c00154. Epub 2021 Jun 1.


RNAs are involved in an enormous range of cellular processes, including gene regulation, protein synthesis, and cell differentiation, and dysfunctional RNAs are associated with disorders such as cancers, neurodegenerative diseases, and viral infections. Thus, the identification of compounds with the ability to bind RNAs and modulate their functions is an exciting approach for developing next-generation therapies. Numerous RNA-binding agents have been reported over the past decade, but the design of synthetic molecules with selectivity for specific RNA sequences is still in its infancy. In this perspective, we highlight recent advances in targeting RNAs with synthetic molecules, and we discuss the potential value of this approach for the development of innovative therapeutic agents.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Antineoplastic Agents / chemistry
  • Antineoplastic Agents / metabolism
  • Antineoplastic Agents / pharmacology
  • Apoptosis / drug effects
  • Drug Discovery*
  • Humans
  • MicroRNAs / antagonists & inhibitors
  • MicroRNAs / metabolism
  • RNA / antagonists & inhibitors
  • RNA / metabolism*
  • RNA Precursors / metabolism
  • RNA Splicing / drug effects
  • RNA, Long Noncoding / antagonists & inhibitors
  • RNA, Long Noncoding / metabolism
  • Small Molecule Libraries / chemistry
  • Small Molecule Libraries / metabolism
  • Small Molecule Libraries / pharmacology


  • Antineoplastic Agents
  • MicroRNAs
  • RNA Precursors
  • RNA, Long Noncoding
  • Small Molecule Libraries
  • RNA