biostudies-literatureUnknown6(3)Zhou CAcute myeloid leukemia (AML) is a common hematological malignancy with overall poor prognosis. Exploring novel targets is urgent and necessary to improve the clinical outcome of relapsed and refractory (RR) AML patients. Through clinical specimens, animal models and cell-level studies, we explored the specific mechanism of 3-hydroxy-3-methylglutaryl coenzyme A synthase 1 (HMGCS1) in AML and the mechanism of targeting HMGCS1 to attenuate cell proliferation, increase chemotherapy sensitivity and improve the occurrence and development of AML. Here, we reveal that HMGCS1 is overexpressed in RR patients and negatively related to overall survival (OS). Knocking out HMGCS1 in AML cells attenuated cell proliferation and increased chemotherapy sensitivity, while stable overexpression of HMGCS1 had Blood science (Baltimore, Md.)e00192https://www.ebi.ac.uk/biostudies/studies/S-EPMC11239175biostudies-literatureTargeting HMGCS1 restores chemotherapy sensitivity in acute myeloid leukemia.PMC11239175Li JZhao LZhang TLiang HZhou CZhan HFang PZeng HDu JSun XHe QfalseTargeting HMGCS1 restores chemotherapy sensitivity in acute myeloid leukemia.Acute myeloid leukemia (AML) is a common hematological malignancy with overall poor prognosis. Exploring novel targets is urgent and necessary to improve the clinical outcome of relapsed and refractory (RR) AML patients. Through clinical specimens, animal models and cell-level studies, we explored the specific mechanism of 3-hydroxy-3-methylglutaryl coenzyme A synthase 1 (HMGCS1) in AML and the mechanism of targeting HMGCS1 to attenuate cell proliferation, increase chemotherapy sensitivity and improve the occurrence and development of AML. Here, we reveal that HMGCS1 is overexpressed in RR patients and negatively related to overall survival (OS). Knocking out HMGCS1 in AML cells attenuated cell proliferation and increased chemotherapy sensitivity, while stable overexpression of HMGCS1 had 2024-01-01T00:00:00Z2024 Jul2025-04-19T01:06:05.058Z2025-04-07T12:05:01.526ZS-EPMC112391753899452510.1097/BS9.0000000000000192