Fig. 4: Moderate Gata2 reduction increases competitiveness of Cebpa-mutant AML.
a Experimental setup for evaluating the effect of Gata2 knockdown, via short hairpin RNA (shRNA) mediated silencing, on Cebpap30/p30 leukemic cells in a competitive in vivo assay. The illustration was created with BioRender.com. b Gata2 mRNA in Cebpap30/p30 leukemic cells prior to transplantation. c Representative flow cytometry profiles of input and output of shControl (no knockdown), shGata2A (low knockdown), and shGata2D (high knockdown). d Competitive advantage of targeting shRNA (GFP+) vs. non-targeting shRNA (YFP+) cells in vivo assessed as by flow cytometry (Control 4, shGata2A 4, shGata2B 4, shGata2C 4, and shGata2D 3 mice). Data are presented as mean±SEM. Data were log-transformed and analyzed by one-way-ANOVA followed by Dunnett’s multiple comparisons correction. e Experimental setup for Gata2 CRISPR/Cas9 mutagenesis in Cebpap30/p30 cells, and outgrowth of heterozygous mutated clones. Percentages of Gata2 mutated clones are indicated. f Growth curve of Cebpap30/p30 clones with Gata2 mutation (Cebpap30/p30Gata2+/MUT, n = 10) or wild type Gata2 (Cebpap30/p30Gata2+/+, n = 3). Data are presented as mean±SEM and analyzed by two-tailed unpaired t-test. Red lines mark individual mutated clones. g Presence or absence of GATA2 mutations (GATA2MUT) in CEBPA double mutated (CEBPADM) AML cases with or without TET2 mutations (TET2MUT) in aggregated data from published cohorts3,4,5,7,8,17 (detailed in Supplemental Table 2a). Data were analyzed by Wilson/Brown binominal test. Source data are provided as a Source Data file.
