GATA2 is overexpressed in acute myeloid leukemia (AML) and loss-of-function mutations have been causally linked to immunodeficiency associated with the myelodysplastic syndromes (MDS), suggesting that an appropriate level of GATA2 activity is a prerequisite for normal hematopoiesis [25]. This evidence concerns the gene GATA2 and acute myeloid leukemia.