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DB2313 is a potent transcription factor PU.1 inhibitor with an apoptosis of 14 nM. DB2313 disrupts the interaction of PU.1 with target gene promoters. DB2313 induces apoptosis of acute myeloid leukemia (AML) cells, and has anticancer effects.

DB2313 treatment leads to a profound decrease in the growth of PU.1 URE acute myeloid leukemia (AML) cells (IC₅₀ of 7.1 μM), while showing little effect on normal hematopoietic cells at similar concentrations. DB2313 treatment leads to a 3.5-fold increase in apoptotic cells in murine PU.1 URE AML cells. DB2313 also leads to a significant decrease in clonogenicity in the second and third rounds of plating and a complete disruption of clonogenic capacity in the fourth and higher rounds of plating.
In AML cells, DB2313 decreases PU.1 occupancy on E2f1, Junb, and Csf1r promoters.

DB2313 (17 mg/kg; i.p.; three times per week; for 3 weeks) treatment decreases leukemia progression and results in increased survival in mice.

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