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miRNA expression in acute myeloid leukaemia: new targets for therapy?

Fletcher, Daniel; Brown, Elliott; Javadala, Julliah; Uysal-Onganer, Pinar; Guinn, Barbara


Daniel Fletcher

Elliott Brown

Julliah Javadala

Pinar Uysal-Onganer


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.


Fletcher, D., Brown, E., Javadala, J., Uysal-Onganer, P., & Guinn, B. (in press). miRNA expression in acute myeloid leukaemia: new targets for therapy?. eJHaem,

Journal Article Type Review
Acceptance Date Mar 31, 2022
Online Publication Date Apr 26, 2022
Deposit Date Jun 8, 2022
Publicly Available Date Jun 8, 2022
Journal eJHaem
Print ISSN 2688-6146
Publisher John Wiley and Sons
Peer Reviewed Peer Reviewed
Keywords Acute myeloid leukaemia (AML); MiRNA; MicroRNA; Stemness; Therapy
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Copyright Statement
© 2022 The Authors. eJHaem published by British Society for Haematology and John Wiley & Sons Ltd.<br /> <br /> This is an open access article under the terms of the Creative Commons Attribution License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited.

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