Discovery of BEND4 as a novel single-gene prognostic marker and therapeutic target for adverse AML
A newly discovered protein reliably identifies aggressive acute myeloid leukemia cases that might respond to targeted drug strategies.
BEND4, a BEN domain-containing protein, is identified as a novel single-gene expression biomarker for adverse-risk AML with RT-qPCR discriminatory performance (91% sensitivity, 81% specificity) superior to existing models, validated in a total AML cohort of nearly 1,700 patients. Functional studies confirm therapeutic targetability through modulation of chemoresistance and pro-survival pathways, positioning BEND4 as a candidate for clinical biomarker development.
What the study was
- Study design
- Transcriptome analysis of primary AML cohorts (discovery n=1338, validation n=350); RT-qPCR threshold derivation; in vitro functional validation; in vivo mouse model
- Population
- AML patients with adverse cytogenetic risk; TCGA and clinical AML cohorts
- Sample size
- 1688
- Category
- Genomics/Precision Medicine
- Maturity
- Exploratory
- Journal
- npj Precision Oncology
Why it surfaced
Novel single-gene AML biomarker with competitive RT-qPCR sensitivity/specificity, validated in two independent cohorts. Score capped at 7 due to mixed human/animal design (requires clinical prospective validation) and absence of clinical trial data.
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