Overall these compounds appear to exert promising anti-leukemic functions primarily by altering stem cell programs, inhibiting proliferation, and restoring myeloid differentiation in AML cells, with increased efficacy for leukemia subtypes carrying MLL and AML1 rearrangements, NPM1 mutations, and erythroid and megakaryoblastic differentiation block (15). The gene discussed is KMT2A; the disease is leukemia.