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Updated: Jun 8, 2026

MicroRNA Amplification and Recognition through Locked-nucleic-acid In situ Hybridization as a Novel Detection and Quantification Method
Published on: October 7, 2025
Targeting microRNAs in cancer: rationale, strategies and challenges
Ramiro Garzon1, Guido Marcucci, Carlo M Croce
1Division of Haematology and Oncology, Department of Medicine, Comprehensive Cancer Center, The Ohio State University, Columbus, Ohio 43221, USA. ramiro.garzon@osumc.edu
Abstract:
MicroRNAs (miRNAs) are evolutionarily conserved small non-coding RNAs that regulate gene expression. Early studies have shown that miRNA expression is deregulated in cancer and experimental data indicate that cancer phenotypes can be modified by targeting miRNA expression. Based on these observations, miRNA-based anticancer therapies are being developed, either alone or in combination with current targeted therapies, with the goal to improve disease response and increase cure rates. The advantage of using miRNA approaches is based on its ability to concurrently target multiple effectors of pathways involved in cell differentiation, proliferation and survival. In this Review, we describe the role of miRNAs in tumorigenesis and critically discuss the rationale, the strategies and the challenges for the therapeutic targeting of miRNAs in cancer.
Insights
MicroRNAs (miRNAs) are small molecules that regulate genes. Targeting miRNA expression offers a promising strategy for developing novel anticancer therapies due to their ability to affect multiple cancer-related pathways.
Area of Science:
- Molecular Biology
- Genetics
- Oncology
Background:
- MicroRNAs (miRNAs) are small, non-coding RNA molecules that play crucial roles in gene expression regulation.
- Aberrant miRNA expression is frequently observed in various types of cancer, contributing to tumorigenesis.
- Experimental evidence suggests that modulating miRNA levels can alter cancer phenotypes.
Purpose of the Study:
- To review the role of miRNAs in cancer development (tumorigenesis).
- To critically evaluate the strategies and challenges associated with developing miRNA-based cancer therapies.
- To discuss the potential of miRNA targeting as a therapeutic approach in oncology.
Main Methods:
- Literature review of existing studies on miRNAs in cancer.
- Analysis of experimental data demonstrating the impact of miRNA modulation on cancer.
- Discussion of current and emerging therapeutic strategies targeting miRNAs.
Main Results:
- miRNAs are key regulators of cellular processes including differentiation, proliferation, and survival.
- Dysregulated miRNAs are implicated in the initiation and progression of cancer.
- Targeting miRNAs can modify cancer phenotypes and offers therapeutic potential.
Conclusions:
- miRNA-based therapies hold promise for cancer treatment, potentially by targeting multiple oncogenic pathways simultaneously.
- Combining miRNA therapeutics with existing treatments may improve patient outcomes and increase cure rates.
- Further research is needed to overcome challenges and optimize miRNA-targeting strategies for clinical application in cancer.
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