The inhibition of BET proteins with preclinical inhibitors, such as JQ1 showed promising results in several studies in AML cell lines and ex vivo patient samples or mouse models, in particular in specific subtypes with MLL rearrangement, or those with mutations in NPM1, FLT3 or IDH2, or EVI1 overexpression (89, 94–97). The gene discussed is RUNX1; the disease is acute myeloid leukemia.