The RNAi- and K1717 (GATA2 inhibitor)-mediated inhibition of GATA2 notably diminished the expression of GATA2 targets—namely, Enah/Vasp-like AML markers, stem-like leukemia markers, and WT1 (Wilms tumor1, implicated in AML)—in KG1a cells and in clinical patients’ AML cells, triggering apoptosis. This evidence concerns the gene GATA2 and acute myeloid leukemia.