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A Perturb-seq map of a differentiation hub reveals synergistic vulnerabilities in KMT2A-rearranged acute myeloid leukemia - PubMed

3 hours ago
  • #acute myeloid leukemia
  • #combination therapy
  • #epigenetics
  • Perturb-seq screening identifies a synergistic epigenetic hub (KAT6A, Menin, DOT1L) that silences a core myeloid differentiation program in KMT2A-rearranged AML.
  • Simultaneous pharmacological inhibition of hub components robustly reactivates the myeloid program, showing synergistic anti-leukemic effects.
  • The myeloid program serves as a predictive biomarker; high baseline activity indicates vulnerability to MEK, AKT, and mTOR inhibitors.