It induced myeloid differentiation and disrupted colony formation in AML cell lines (MOLM13, TF-1, U937, MV4–11, HL-60 and NB4) and patient cells, while showing no effects on normal hematopoietic cells and shows therapeutic efficacy in vivo across multiple xenograft and syngeneic mouse models of AML driven by the oncogene MLL-AF9 [168]. The gene discussed is MLLT3; the disease is acute myeloid leukemia.