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December 8, 2025BloodOpen Access

NUP98-rearranged pediatric AML is sustained by a conserved leukemia stem cell program with SPNS2/3 as a targetable vulnerability

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Authors

EDElisabeth DenbyRZRun ZhangTHTingting Hu

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Overview

Findings reveal leukemia stem cell vulnerabilities in NUP98-rearranged AML, suggesting SPNS2/3 as a therapeutic target.

Key Points

  • This research aims to identify targetable vulnerabilities in NUP98-rearranged pediatric AML through genetic and molecular profiling.
  • Developed genetically diverse AML models using induced pluripotent stem cells and CRISPR/Cas9 gene editing.
  • Used CITE-seq to characterize leukemia stem cell populations and their gene expression signatures.
  • Examined therapeutic responses to SPNS2/3 inhibition in vitro and in vivo.
  • Identified specific leukemia stem cell populations in NUP98-rearranged AML models.
  • Demonstrated that SPNS2/3 are potential therapeutic targets, impairing AML growth and improving survival in vivo.
  • Explored distinct gene expression signatures corresponding to different genetic subclasses of AML.

Cite This Study

Denby et al. (2025) studied this question.

synapsesocial.com/papers/693624d74fa91c937236d110https://doi.org/10.1182/blood-2025-5002
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