PLZF-RARα direct transcriptional target genes are associated with an APL-specific phenotype. Gene expression profiles of 22 APL (PLZF-RARα [n = 4] and PML-RARα [n = 18]) and 99 AML patients were normalized by GC-RMA, and GSEA was used to generate a ranked list of genes distinguishing these phenotypes (red = up-regulated in APL vs AML blue = down-regulated in APL vs AML). The distribution of PLZF-RARα direct targets (1672 genes) within this ranked gene list was calculated and expressed as number of genes/decile. PLZF-RARα–bound and regulated genes are highly enriched in deciles 8 to 10, representing genes down-regulated in APL versus AML (●, P < .001), whereas genes from a randomly selected dataset were not significantly enriched in these quartiles (○, .01 < P < .1).