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Deciphering molecular heterogeneity in pediatric AML using a cancer vs. normal transcriptomic approach.

Barbara DepreterBarbara De MoerlooseKarl VandepoeleAnne UyttebroeckAn Van DammeEva TerrasBarbara DenysLaurence DedekenMarie-Françoise DresseJutte Van der Werff Ten BoschMattias HofmansJan PhilippéTim Lammens
Published in: Pediatric research (2020)
Novel transcriptional targets were discovered showing a significant differential expression in LSCs and blasts from pedAML patients compared to their normal counterparts from healthy controls. Deregulated pathways, including immune and metabolic dysregulation, were addressed for the first time in children, offering a deeper understanding of the molecular pathogenesis. These novel targets have the potential of acting as biomarkers for risk stratification, follow-up, and targeted therapy. Multiple LSC-downregulated targets endow tumor suppressor roles in other cancer entities, and further investigation whether hypomethylating therapy could result into LSC eradication in pedAML is warranted.
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