Small-molecule Approaches Toward the Targeting of Oncogenic miRNAs: Roadmap for the Discovery of RNA Modulators

Future Med Chem. 2016 May;8(7):803-16. doi: 10.4155/fmc-2016-0018. Epub 2016 May 5.


miRNAs are a recently discovered class of small noncoding RNAs implicated in the regulation of gene expression. The deregulation of miRNAs levels has been linked to the development of various cancers where oncogenic miRNAs are overexpressed and tumor suppressor miRNAs are underexpressed. Here we report the three main strategies developed in order to discover small-molecule drugs able to selectively interfere with oncogenic miRNAs: the high throughput screening of large libraries of compounds, the focused screening of small libraries of molecules that are known to be able to interact with RNA thus being supposed modulators of miRNAs pathway and the design of small molecules based on the secondary structure of targeted RNA and/or three-dimensional structure of enzymes involved in miRNAs pathway.

Keywords: inhibitor; oncogenic miRNA; small molecule.

Publication types

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

MeSH terms

  • Antineoplastic Agents / chemistry
  • Antineoplastic Agents / pharmacology
  • Antineoplastic Agents / therapeutic use*
  • Drug Screening Assays, Antitumor
  • Gene Expression
  • High-Throughput Screening Assays
  • Humans
  • Ligands
  • MicroRNAs / antagonists & inhibitors
  • MicroRNAs / metabolism*
  • Molecular Targeted Therapy
  • Neoplasms / drug therapy*
  • Neoplasms / genetics
  • Neoplasms / metabolism
  • Small Molecule Libraries / chemistry
  • Structure-Activity Relationship


  • Antineoplastic Agents
  • Ligands
  • MicroRNAs
  • Small Molecule Libraries