Table 2.

Selected, clinically validated target genes of AML1/ETO (identified with the use of unbiased screening approaches).

Target gene	Function	Biological process	Reference
Upregulated
SOX4, IL-17RB, CD200, and JUP (γ-catenin)	Transcription factor, cytokine receptor, anti-inflammatory signal, Adherens junction	Transcriptional regulation, inflammation, cell adhesion	Tonks et al. [61]
p21waf1 (CDKN1A)	Cyclin-dependent kinase inhibitor	Cell cycle, stem cell maintenance	Berg et al. [59], Peterson et al. [60]
BCL2a	Anti-apoptotic signal	Apoptosis	Martens et al. [66], Klampfer et al. [119]
FOXO1	Transcription factor	Apoptosis, stem cell maintenance	Lin et al. [120]
GFI1	Transcriptional repressor	Transcriptional regulation, G1/S-transition, oncogene	Marneth et al. [121]
TRKA (NTRK1)	MAPK pathway activation, protein kinase	Neuronal development, myeloid differentiation	Mulloy et al. [122]
ZFP36L1 (ERF-1, TIS11b)	Polypeptide chain release factor	mRNA translation	Shimada et al. [123]
ARG2, MT2A	Arginine metabolism, metal homeostasis	Immune response, oxidative Stress	Shia et al. [39]
CD48	NK-cell mediated immunosurveillance	Adaptive immune response, leukocyte migration	Wang et al. [37]
CSF3R	Cytokine receptor	Regulation of hematopoiesis	Shimizu et al. [62]
PAX5	Transcription factor	B-cell maturation	Tiacci et al. [124]
POU4F1	Transcription factor	Transcriptional regulation, B-lymphoid expression	Fortier et al. [125], Dunne et al. [126]
VLA4 (ITGA4)	Cell adhesion and migration	Leukocyte trafficking, Regulation of hematopoiesis	Ponnusamy et al. [127]
Downregulated
IL-3	Cytokine	Regulation of hematopoiesis	Buchi et al. [46]
CSF2	Cytokine	Regulation of hematopoiesis	Frank et al. [63]
CCL3	Chemokine ligand	Chemotaxis, immune response	Bristow and Shore [128]
CEBPA	Transcription factor	Regulation of hematopoiesis	Koschmieder et al. [56] Pabst et al. [57]
LAT2	Adaptor molecule	Regulation of hematopoiesis	Fliegauf et al. [47] Duque-Afonso et al. [49, 52]; Essig [50]
p14ARF (CDKN2A)b	Cyclin-dependent kinase inhibitor	Cell cycle, G1/S-transition, stem cell maintenance	Linggi et al. [64]
RASSF2	K-RAS-specific effector protein	Rac GTPase activation, Rac-mediated signal transduction	Stoner et al. [129]
Lysozyme (LYZ)	Bacteriolytic enzyme	Antimicrobial humoral response, myeloid differentiation	Claus et al. [130]
OGG1	DNA repair enzyme	Response to oxidative stress	Liddiard et al. [131]
PSGL1 (SELPLG)	Cell adhesion and migration	Leukocyte trafficking, regulation of hematopoiesis	Ponnusamy et al. [132]
NF1	GTPase-activating protein	Ras signal transduction	Yang et al. [133]
miR 144/451	Posttranscriptional regulation	Erythroid differentiation	Kohrs et al. [134]
SPI1 (PU.1)	Transcription factor	Regulation of hematopoiesis	Vangala et al. [135]

^aIndividual studies have demonstrated downregulation of BCL2 in leukemic cell lines [136].

^bReferring to the alternate open reading frame (ARF) which does not function as a CDK4/6 inhibitor.