High-throughput luciferase reporter assay for small-molecule inhibitors of microRNA function

J Biomol Screen. 2012 Jul;17(6):822-8. doi: 10.1177/1087057112439606. Epub 2012 Mar 12.

Abstract

MicroRNAs (miRNAs) are endogenous, single-stranded, noncoding RNAs of 21 to 23 nucleotides that regulate gene expression, typically by binding the 3' untranslated regions of target messenger RNAs. It is estimated that miRNAs are involved in the regulation of 30% of all genes and almost every genetic pathway. Recently, the misregulation of miRNAs has been linked to various human diseases including cancer and viral infections, identifying miRNAs as potential targets for drug discovery. Thus, small-molecule modifiers of miRNAs could serve as lead structures for the development of new therapeutic agents and be useful tools in the elucidation of detailed mechanisms of miRNA function. As a result, we have developed a high-throughput screen for potential small-molecule regulators of the liver-specific microRNA miR-122, which is involved in hepatocellular carcinoma development and hepatitis C virus infection. Our small-molecule screen employs a Huh7 human hepatoma cell line stably transfected with a Renilla luciferase sensor for endogenous miR-122. The assay was optimized and validated using an miR-122 antisense agent and a previously identified small-molecule miR-122 inhibitor. The described reporter assay will enable the high-throughput screening of small-molecule miR-122 inhibitors and can be readily extended to other miRNAs.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Validation Study

MeSH terms

  • Antineoplastic Agents / pharmacology*
  • Antiviral Agents / pharmacology*
  • Carcinoma, Hepatocellular / genetics
  • Carcinoma, Hepatocellular / metabolism
  • Cell Line, Tumor
  • Gene Expression
  • Genes, Reporter
  • Hepacivirus / metabolism
  • Hepatitis C / genetics
  • Hepatitis C / metabolism
  • High-Throughput Screening Assays / methods*
  • Humans
  • Liver Neoplasms / genetics
  • Liver Neoplasms / metabolism
  • Luciferases, Renilla / genetics
  • Luciferases, Renilla / metabolism*
  • MicroRNAs / antagonists & inhibitors*
  • MicroRNAs / metabolism
  • Oligonucleotides, Antisense
  • Small Molecule Libraries / pharmacology*
  • Transfection

Substances

  • Antineoplastic Agents
  • Antiviral Agents
  • MIRN122 microRNA, human
  • MicroRNAs
  • Oligonucleotides, Antisense
  • Small Molecule Libraries
  • Luciferases, Renilla