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Updated: Sep 23, 2025

Investigation of the Transcriptional Role of a RUNX1 Intronic Silencer by CRISPR/Cas9 Ribonucleoprotein in Acute Myeloid Leukemia Cells
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Deciphering the Non-Coding RNA Landscape of Pediatric Acute Myeloid Leukemia

Jolien Vanhooren1,2,3, Laurens Van Camp1,2,3, Barbara Depreter4

  • 1Department of Pediatric Hematology-Oncology and Stem Cell Transplantation, Ghent University Hospital, 9000 Ghent, Belgium.

Cancers
|May 14, 2022
PubMed

Insights

This study identifies key non-coding RNAs in pediatric acute myeloid leukemia (pedAML) stem cells. Understanding these molecules may lead to better treatments for children with pedAML, improving survival rates.

Area of Science:

  • Oncology
  • Molecular Biology
  • Genetics

Background:

  • Pediatric acute myeloid leukemia (pedAML) remains a significant challenge, with 20-30% of children experiencing relapse or treatment toxicity.
  • Leukemic stem cells (LSCs) are implicated in pedAML relapse, necessitating their molecular characterization for improved therapies.
  • Non-coding RNAs, including long non-coding RNAs (lncRNAs) and microRNAs (miRNAs), are increasingly recognized for their roles in disease, yet data in pedAML is limited.

Purpose of the Study:

  • To comprehensively analyze non-coding RNA expression in pediatric acute myeloid leukemia (pedAML) subpopulations.
  • To identify differentially expressed lncRNAs and miRNAs in leukemic stem cells (LSCs) compared to normal counterparts.
  • To investigate the regulatory roles and functional implications of identified lncRNAs in pedAML pathogenesis.

Main Methods:

  • Exploration of lncRNA (n=30,168) and miRNA (n=627) expression profiles in pedAML LSCs, leukemic blasts (L-blasts), and their normal counterparts.
  • Assessment of regulatory interactions between differentially expressed lncRNAs in LSCs and miRNAs.
  • Prediction of functional relevance of upregulated lncRNAs in LSCs using gene set enrichment analysis of correlated protein-coding genes.

Main Results:

  • Cataloged differential expression of numerous lncRNAs and miRNAs in pedAML LSCs and L-blasts.
  • Identified potential regulatory networks involving lncRNAs targeting miRNAs within pedAML subpopulations.
  • Predicted functional roles for upregulated lncRNAs in LSCs, offering insights into disease mechanisms.

Conclusions:

  • This study provides a valuable catalog of non-coding RNAs potentially involved in pediatric acute myeloid leukemia (pedAML) pathogenesis.
  • The findings highlight the importance of non-coding RNAs in leukemic stem cells and their potential as therapeutic targets.
  • This research lays the groundwork for future translational studies aimed at developing novel pedAML treatments.

Keywords:
cellular subpopulationsnon-coding RNApediatric AML

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