Targeting MicroRNAs with Small Molecules

Kisanet Tadesse, Raphael I. Benhamou*

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review


MicroRNAs (miRs) have been implicated in numerous diseases, presenting an attractive target for the development of novel therapeutics. The various regulatory roles of miRs in cellular processes underscore the need for precise strategies. Recent advances in RNA research offer hope by enabling the identification of small molecules capable of selectively targeting specific disease-associated miRs. This understanding paves the way for developing small molecules that can modulate the activity of disease-associated miRs. Herein, we discuss the progress made in the field of drug discovery processes, transforming the landscape of miR-targeted therapeutics by small molecules. By leveraging various approaches, researchers can systematically identify compounds to modulate miR function, providing a more potent intervention either by inhibiting or degrading miRs. The implementation of these multidisciplinary approaches bears the potential to revolutionize treatments for diverse diseases, signifying a significant stride towards the targeting of miRs by precision medicine.

Original languageAmerican English
Article number17
JournalNon-coding RNA
Issue number2
StatePublished - Apr 2024

Bibliographical note

Publisher Copyright:
© 2024 by the authors.


  • microRNA
  • small molecules
  • targeted degradation
  • therapeutics


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