MicroRNAs and acute myeloid leukemia: therapeutic implications and emerging concepts

Jared A. Wallace and Ryan M. O'Connell


Acute myeloid leukemia (AML) is a deadly hematologic malignancy characterized by the uncontrolled growth of immature myeloid cells. Over the past several decades we have learned a tremendous amount regarding the genetic aberrations that govern disease development in AML. Among these are genes that encode non-coding RNAs, including the microRNA (miRNA) family. miRNAs are evolutionary conserved small non-coding RNAs that display important physiological effects through their post-transcriptional regulation of messenger RNA (mRNA) targets. Over the past decade, studies have identified miRNAs as playing a role in nearly all aspects of AML disease development, including cellular proliferation, survival, and differentiation. These observations have led to the study of miRNAs as biomarkers of disease, and efforts to therapeutically manipulate miRNAs to improve disease outcome in AML are ongoing. While much has been learned regarding the importance of miRNAs in AML disease initiation and progression, there are many unanswered questions and emerging facets of miRNA biology that add complexity to their roles in AML. Moving forward, answers to these questions will provide a greater level of understanding of miRNA biology and critical insights into the many translational applications for these small regulatory RNAs in AML.

  • Submitted October 4, 2016.
  • Accepted July 24, 2017.