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Updated: May 17, 2026
09:06
MicroRNA Amplification and Recognition through Locked-nucleic-acid In situ Hybridization as a Novel Detection and Quantification Method
Published on: October 7, 2025
Discovering the first microRNA-targeted drug
Morten Lindow1, Sakari Kauppinen
1Department of Biology, The Bioinformatics Centre, University of Copenhagen, DK-2200 Copenhagen, Denmark.
The Journal of Cell Biology
|October 31, 2012
Summary
MicroRNAs regulate biological processes and disease. Miravirsen, a novel therapeutic targeting microRNA-122, is in clinical trials for hepatitis C virus infection.
Area of Science:
- Molecular Biology
- Genetics
- Pharmacology
Background:
- MicroRNAs (miRNAs) are crucial post-transcriptional regulators involved in cellular processes.
- Dysregulation of miRNAs contributes to the development of various human diseases.
- miRNA-based therapeutics represent a promising avenue for drug discovery.
Purpose of the Study:
- To describe the discovery and development of miravirsen, a novel therapeutic agent.
- To highlight miravirsen's mechanism of action targeting miRNA-122.
- To present the current status of miravirsen in clinical trials for hepatitis C virus (HCV) infection.
Main Methods:
- Discovery of miravirsen, a locked nucleic acid (LNA)-modified antisense oligonucleotide.
- Targeting of the liver-expressed miRNA-122.
- Evaluation in preclinical and clinical studies for HCV treatment.
Main Results:
- Miravirsen was identified as a potent inhibitor of miRNA-122.
- The drug has advanced to Phase 2 clinical trials for HCV infection.
- This represents a significant step in miRNA-based therapeutic development.
Conclusions:
- Miravirsen demonstrates the therapeutic potential of targeting specific miRNAs.
- Antisense oligonucleotide technology is effective for miRNA modulation.
- Further clinical development is ongoing for this innovative HCV treatment.
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