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Article

Flavopiridol restores granulopoiesis in experimental models of severe congenital neutropenia.

Molecular therapy : the journal of the American Society of Gene Therapy - 4 Jun 2025

Nasri Masoud, Dannenmann Benjamin, Doll Larissa, Findik Betül, Bernhard Franka, Kandabarau Sergey, Klimiankou Maksim, Gawaz Meinrad, Lengerke Claudia, Zeidler Cornelia, Welte Karl, Skokowa Julia

Abstract excerpt

Severe congenital neutropenia (CN) patients require life-long treatment with recombinant human granulocyte colony-stimulating factor (rhG-CSF), but some show no response. We sought to establish a therapy for CN that targets signaling pathways causing maturation arrest of granulocytic progenitors. We developed an isogenic induced pluripotent stem cell (iPSC) in vitro model of CN associated with ELANE mutations...

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Identifiers

PMID: 39653038
DOI: 10.1016/j.ymthe.2024.10.031
PMCID: PMC12172196

Genes

ELANE
CD34

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 2 Sept 2026.

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