Article

Cholesterol Biosynthesis is a Targetable Vulnerability of <i>CEBPA</i> -mutant Acute Myeloid Leukemia

2026-02-20

Abstract excerpt

Bi-allelic CEBPA mutations occur in 5-15% of acute myeloid leukemia (AML) patients. The precise molecular consequences of CEBPA mutations, especially in combination with frequently co-occurring mutations in TET2 , WT1 , and GATA2, remain incompletely understood. Here, we present a robust human model of CEBPA -mutant AML through gene editing of healthy bone marrow-derived hematopoietic stem cells. Loss of the...

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Literature Corpus work: d1cbeabe-bb72-51f1-a251-fc69fd648c37
DOI: 10.64898/2026.02.20.706425

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