AML1/RUNX1 functions as a cytoplasmic attenuator of NF-κB signaling in the repression of myeloid tumors

Blood. 2011 Dec 15;118(25):6626-37. doi: 10.1182/blood-2010-12-326710. Epub 2011 Oct 21.

Abstract

Functional deregulation of transcription factors has been found in many types of tumors. Transcription factor AML1/RUNX1 is one of the most frequent targets of chromosomal abnormalities in human leukemia and altered function of AML1 is closely associated with malignant transformation of hematopoietic cells. However, the molecular basis and therapeutic targets of AML1-related leukemia are still elusive. Here, we explored immediate target pathways of AML1 by in vitro synchronous inactivation in hematopoietic cells. We found that AML1 inhibits NF-κB signaling through interaction with IκB kinase complex in the cytoplasm. Remarkably, AML1 mutants found in myeloid tumors lack the ability to inhibit NF-κB signaling, and human cases with AML1-related leukemia exhibits distinctly activated NF-κB signaling. Furthermore, inhibition of NF-κB signaling in leukemic cells with mutated AML1 efficiently blocks their growth and development of leukemia. These findings reveal a novel role for AML1 as a cytoplasmic attenuator of NF-κB signaling and indicate that NF-κB signaling is one of the promising therapeutic targets of hematologic malignancies with AML1 abnormality.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Acute Disease
  • Animals
  • Antineoplastic Agents / pharmacology
  • Blotting, Western
  • Boronic Acids / pharmacology
  • Bortezomib
  • Cell Line, Tumor
  • Cell Transformation, Neoplastic / genetics
  • Core Binding Factor Alpha 2 Subunit / genetics
  • Core Binding Factor Alpha 2 Subunit / metabolism
  • Core Binding Factor Alpha 2 Subunit / physiology*
  • Cytoplasm / metabolism
  • Female
  • Gene Expression Regulation, Leukemic
  • HEK293 Cells
  • Humans
  • I-kappa B Kinase / genetics
  • I-kappa B Kinase / metabolism
  • Leukemia, Myeloid / drug therapy
  • Leukemia, Myeloid / metabolism*
  • Leukemia, Myeloid / pathology
  • Mice
  • Mice, Inbred C57BL
  • Mice, Inbred NOD
  • Mice, Knockout
  • Mice, SCID
  • Mutation
  • NF-kappa B / genetics
  • NF-kappa B / metabolism*
  • Protein Binding
  • Pyrazines / pharmacology
  • Signal Transduction / drug effects
  • Signal Transduction / genetics
  • Signal Transduction / physiology*
  • Xenograft Model Antitumor Assays

Substances

  • Antineoplastic Agents
  • Boronic Acids
  • Core Binding Factor Alpha 2 Subunit
  • NF-kappa B
  • Pyrazines
  • Runx1 protein, mouse
  • Bortezomib
  • I-kappa B Kinase