A novel murine model of myeloproliferative disorders generated by overexpression of the transcription factor NF-E2

J Exp Med. 2012 Jan 16;209(1):35-50. doi: 10.1084/jem.20110540. Epub 2012 Jan 9.

Abstract

The molecular pathophysiology of myeloproliferative neoplasms (MPNs) remains poorly understood. Based on the observation that the transcription factor NF-E2 is often overexpressed in MPN patients, independent of the presence of other molecular aberrations, we generated mice expressing an NF-E2 transgene in hematopoietic cells. These mice exhibit many features of MPNs, including thrombocytosis, leukocytosis, Epo-independent colony formation, characteristic bone marrow histology, expansion of stem and progenitor compartments, and spontaneous transformation to acute myeloid leukemia. The MPN phenotype is transplantable to secondary recipient mice. NF-E2 can alter histone modifications, and NF-E2 transgenic mice show hypoacetylation of histone H3. Treatment of mice with the histone deacetylase inhibitor (HDAC-I) vorinostat restored physiological levels of histone H3 acetylation, decreased NF-E2 expression, and normalized platelet numbers. Similarly, MPN patients treated with an HDAC-I exhibited a decrease in NF-E2 expression. These data establish a role for NF-E2 in the pathophysiology of MPNs and provide a molecular rationale for investigating epigenetic alterations as novel targets for rationally designed MPN therapies.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Blood Cell Count
  • Blood Cells / metabolism
  • Cell Differentiation / genetics
  • Chromatin / metabolism
  • Disease Models, Animal*
  • Disease Progression
  • Gene Expression
  • Gene Expression Regulation
  • Hematopoietic Stem Cells / cytology
  • Hematopoietic Stem Cells / metabolism
  • Histone Deacetylase Inhibitors / pharmacology
  • Histone Deacetylase Inhibitors / therapeutic use
  • Humans
  • Leukemia / metabolism
  • Leukemia / pathology
  • Mice
  • Mice, Transgenic*
  • Myeloproliferative Disorders / drug therapy
  • Myeloproliferative Disorders / genetics*
  • Myeloproliferative Disorders / metabolism
  • NF-E2 Transcription Factor / genetics*
  • Phenotype

Substances

  • Chromatin
  • Histone Deacetylase Inhibitors
  • NF-E2 Transcription Factor