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A genetic disorder reveals a hematopoietic stem cell regulatory network co-opted in leukemia

15 Dec 2022

Abstract excerpt

The molecular regulation of human hematopoietic stem cell (HSC) maintenance is therapeutically important, but limitations in experimental systems and interspecies variation have constrained our knowledge of this process. Here, we have studied a rare genetic disorder due to MECOM haploinsufficiency, characterized by an early-onset absence of HSCs in vivo. By generating a faithful model of this disorder in primary...

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PMID: 36522544
DOI: 10.1038/s41590-022-01370-4

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