In this study, we explored the functional role of GATA1s in DS AMkL biology and therapy using lentivirus shRNA to knockdown GATA1 in the DS AMkL cell line, CMK, which expresses only GATA1s and no wild-type GATA1.[17] Our results suggest that GATA1s has unique functions in facilitating DS leukemogenesis and in modulating therapeutic responses by repressing differentiation towards the megakaryocytic lineage, and by promoting proliferation and survival, potentially through regulating expression of Bcl-2 and other relevant genes. The gene discussed is GATA1; the disease is Dravet syndrome.