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BTSA1 is an activator of Bax.1 It binds to the Bax N-terminal activation site, also known as the trigger site (IC50 = 250 nM), and induces Bax translocation membrane permeabilization in liposomes when used at concentrations ranging from 100 to 400 nM. BTSA1 (0.1-1 µM) induces apoptosis in various human and mouse acute myeloid leukemia (AML) cell lines. In vivo, BTSA (10 mg/kg) reduces bone marrow infiltration and promotes apoptosis in bone marrow infiltrates in a MOLM-13 AML mouse xenograft model.
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1. Direct activation of BAX by BTSA1 overcomes apoptosis resistance in acute myeloid leukemia. Cancer Cell 32(4), 490-505 (2017).