Comprehensive Transcriptome Profiling of Cryptic <i>CBFA2T3–GLIS2</i> Fusion–Positive AML Defines Novel Therapeutic Options: A COG and TARGET Pediatric AML Study

The <i>CBFA2T3-GLIS2</i> fusion defines a highly refractory entity limited to infants that appears to be sufficient for malignant transformation. Transcriptome profiling elucidated several highly targetable genes and pathways, including the identification of CD56, providing a highly plausible target for therapeutic intervention.