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Updated: May 10, 2026

MicroRNA Amplification and Recognition through Locked-nucleic-acid In situ Hybridization as a Novel Detection and Quantification Method
Published on: October 7, 2025
Small molecules modulating biogenesis or processing of microRNAs with therapeutic potentials
1School of Chemistry and Chemical Engineering, State Key Lab of Analytical Chemistry for Life Science, Nanjing University, China.
Abstract:
MicroRNAs (miRNAs) are single-stranded non-coding RNAs with the ability to regulate gene expression at post-transcriptional level. Typically, miRNAs function by binding to the 3' untranslated regions (UTR) of target mRNAs, leading to the degradation or repressed expression of target genes. It is estimated that miRNAs are involved in almost every genetic pathway and the regulation of miRNAs plays important roles in physiological and pathological processes. Small molecules that can regulate miRNAs have great potential as probes to explore miRNAs-mediated regulatory network. Small-molecule regulators of disease-related miRNAs also hold the potential as novel therapeutic agents. Based on the screening systems developed in recent years, several small-molecule regulators have been identified as specific or universal regulators of miRNAs. Therapeutic potentials of these small molecules have also been demonstrated. A general review on the reported small molecules modulating the biogenesis or function of miRNAs will be presented in this paper, with emphasis on the screening methods, proposed mechanism of action and the therapeutic potentials of these small molecules.
Insights
Small molecules can regulate microRNAs (miRNAs), which are key gene regulators. This review covers small-molecule miRNA modulators, their screening, mechanisms, and therapeutic potential for diseases.
Area of Science:
- Molecular Biology
- Genetics
- Pharmacology
Background:
- MicroRNAs (miRNAs) are crucial regulators of gene expression at the post-transcriptional level.
- miRNAs influence nearly all genetic pathways and are vital in physiological and pathological processes.
- Dysregulation of miRNAs is implicated in various diseases, highlighting their therapeutic relevance.
Purpose of the Study:
- To review small molecules that modulate microRNA (miRNA) biogenesis or function.
- To emphasize screening methods for identifying these small-molecule regulators.
- To discuss proposed mechanisms of action and therapeutic potentials of identified compounds.
Main Methods:
- Literature review of studies reporting small-molecule regulators of miRNAs.
- Analysis of screening systems used for identifying miRNA modulators.
- Evaluation of proposed mechanisms and demonstrated therapeutic applications.
Main Results:
- Several small-molecule regulators of miRNAs have been identified using recent screening systems.
- These molecules act as specific or universal regulators of miRNA activity.
- Therapeutic potential of these small molecules in disease contexts has been demonstrated.
Conclusions:
- Small molecules offer promising tools for probing miRNA-mediated networks.
- Disease-related miRNA modulators hold significant potential as novel therapeutic agents.
- Further research into small-molecule miRNA regulators could lead to new treatment strategies.
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