Severe congenital neutropenia (CN) is a pre-leukemic bone marrow failure syndrome that can evolve to acute myeloid leukemia (AML). Certain genetic mutations are frequently observed in CN patients, although how they drive the transition from CN to AML (CN/AML) is unclear. In this study, the researchers established an in vitro model of leukemia evolution in CN using patient-derived iPSCs and CRISPR-Cas9-mediated introduction of gene mutations. Using this model, they described the upregulation of the gene BAALC as a key leukemogenic event and identiﬁed a small-molecule targeting CN/AML blasts without affecting healthy cells. This strategy could be applied to treat CN/AML or some de novo AML patients.
iPSC modeling of stage-specific leukemogenesis reveals BAALC as a key oncogene in severe congenital neutropenia
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