This study reveals a mechanism whereby CBFβ-SMMHC drives leukemia maintenance and suggests that inhibitors targeting chromatin activity may prove effective in inv(16) leukemia therapy.
Previous studies showed that the interaction between CBFβ-smooth muscle myosin heavy chain (SMMHC; encoded by <i>CBFB-MYH11</i>) and RUNX1 plays a critical role in the pathogenesis of this leukemia.
Although knock-in of CBFB-MYH11 is not sufficient to cause disease, expression increases the incidence of leukemia when combined with cooperating events.
Although CBFB forms a core-binding factor transcriptional complex with RUNX1, previous analyses have excluded the CBFB gene as the leukemia-predisposing gene in these families.