In AML, recent detailed mechanistic studies have identified DOT1L as a vulnerability of MLL-rearranged leukemia (Bernt et al., 2011), and both a mechanistic and an RNAi-based epigenetics-focused screen identified BRD4 as a therapeutic target against AMLs of different genotypes (Dawson et al., 2011, Zuber et al., 2011). The gene discussed is DOT1L; the disease is acute myeloid leukemia.