It seems that the strategy of inhibiting SMARCA5 in AML to block leukemogenesis becomes even more vital as shown recently using inhibitors of SMARCA5 (ED2-AD101) that target the HELICc-DExx domain to release the terminal AML cells into differentiation while sparing normal hematopoiesis in preclinical animal models [28]. The gene discussed is GJB6; the disease is acute myeloid leukemia.