Small-Molecule Regulation of MicroRNA Function

  • Colleen M. Connelly
  • Alexander DeitersEmail author


MicroRNAs (miRNAs) are single-stranded noncoding RNAs of 21–23 nucleotides, which regulate the expression of genes by binding to the 3′ untranslated regions of target messenger RNAs (mRNAs). MicroRNAs down-regulate gene expression by either inhibiting translation or accelerating the degradation of the mRNA. It is estimated that miRNAs are involved in the regulation of about 30 % of all genes and almost every genetic pathway, making miRNAs an important class of gene regulators. Variations in miRNA expression are involved in many human diseases including cancer, immune disorders, diabetes, and cardiovascular diseases. Thus, small molecule modifiers of miRNA function have potential as new therapeutic agents, as probes for the elucidation of detailed mechanisms of miRNA function and regulation, and as tools for the discovery of new targets for the treatment of human diseases. A variety of different assay systems have been developed and used in the discovery of small molecule modifiers of miRNA function. Identified small molecules regulate the miRNA pathway in either a general or a miRNA-specific fashion. The discovery and development of these molecules demonstrates that the miRNA pathway represents a feasible small molecule target. Several of these small molecules have also shown therapeutic potential in cell based experiments, supporting the idea that modifiers of miRNA function could lead to the identification of new drugs.


Huh7 Cell RNAi Pathway miRNA Function miRNA Pathway Luciferase Signal 
These keywords were added by machine and not by the authors. This process is experimental and the keywords may be updated as the learning algorithm improves.


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© Springer Science+Business Media Dordrecht 2013

Authors and Affiliations

  1. 1.Department of ChemistryNorth Carolina State UniversityRaleighUSA

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