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Published on: June 6, 2025
microRNA expression in acute myeloid leukaemia: New targets for therapy?
Daniel Fletcher1, Elliott Brown1, Julliah Javadala1
1Department of Biomedical Sciences University of Hull Hull, UK.
Abstract:
Recent studies have shown that short non-coding RNAs, known as microRNAs (miRNAs) and their dysregulation, are implicated in the pathogenesis of acute myeloid leukaemia (AML). This is due to their role in the control of gene expression in a variety of molecular pathways. Therapies involving miRNA suppression and replacement have been developed. The normalisation of expression and the subsequent impact on AML cells have been investigated for some miRNAs, demonstrating their potential to act as therapeutic targets. Focussing on miRs with therapeutic potential, we have reviewed those that have a significant impact on the aberrant biological processes associated with AML, and crucially, impact leukaemic stem cell survival. We describe six miRNAs in preclinical trials (miR-21, miR-29b, miR-126, miR-181a, miR-223 and miR-196b) and two miRNAs that are in clinical trials (miR-29 and miR-155). However none have been used to treat AML patients and greater efforts are needed to develop miRNA therapies that could benefit AML patients in the future.
Insights
MicroRNAs (miRNAs) are key in acute myeloid leukaemia (AML) development. While some miRNAs show therapeutic promise for AML stem cells, further research is needed for clinical application.
Area of Science:
- Oncology
- Molecular Biology
- RNA Therapeutics
Background:
- MicroRNAs (miRNAs) are short non-coding RNAs regulating gene expression.
- miRNA dysregulation is implicated in the pathogenesis of acute myeloid leukaemia (AML).
- miRNAs play a role in controlling molecular pathways crucial for AML development.
Purpose of the Study:
- To review microRNAs with therapeutic potential in AML.
- To focus on miRNAs impacting aberrant biological processes and leukaemic stem cell survival in AML.
- To assess the current status of miRNA-based therapies for AML.
Main Methods:
- Literature review of studies on miRNAs in AML.
- Identification of miRNAs investigated in preclinical and clinical trials for AML.
- Analysis of the impact of specific miRNAs on AML pathogenesis and leukaemic stem cell survival.
Main Results:
- Six miRNAs (miR-21, miR-29b, miR-126, miR-181a, miR-223, miR-196b) are in preclinical AML trials.
- Two miRNAs (miR-29, miR-155) are in clinical AML trials.
- No miRNA-based therapies are currently approved for AML treatment.
Conclusions:
- Certain miRNAs demonstrate significant potential as therapeutic targets for AML.
- Targeting miRNAs could normalize gene expression and impact AML cell behaviour.
- Further development is essential to translate miRNA therapies into effective treatments for AML patients.
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