Somatic inactivation of Nf1 in hematopoietic cells results in a progressive myeloproliferative disorder

Blood. 2004 Jun 1;103(11):4243-50. doi: 10.1182/blood-2003-08-2650. Epub 2004 Feb 24.

Abstract

The NF1 tumor suppressor gene encodes a guanosine triphosphotase (GTPase)-activating protein that negatively regulates Ras signaling and is inactivated in a subset of juvenile myelomonocytic leukemias (JMMLs). Adoptive transfer of fetal liver cells from Nf1 mutant mice models JMML; however, this system has important limitations as a platform for performing biologic and preclinical studies. We have exploited the interferon-inducible Mx1-Cre transgene to ablate a conditional mutant Nf1 allele in hematopoietic cells. Somatic inactivation of Nf1 induces a myeloproliferative disorder with 100% penetrance that is associated with a sub-acute clinical course, tissue infiltration by myeloid cells, hypersensitivity to granulocyte-macrophage colony stimulating factor, hyperproliferation, and resistance to apoptosis. These Mx1-Cre, Nf1(flox/flox) mice establish a tractable experimental model for testing therapeutics and for identifying mutations that cooperate with hyperactive Ras in myeloid leukemogenesis.

Publication types

  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, Non-P.H.S.
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Adoptive Transfer
  • Animals
  • Apoptosis / immunology
  • Cell Division / immunology
  • Disease Models, Animal*
  • GTP-Binding Proteins / genetics
  • Integrases / genetics
  • Leukocytes / pathology
  • Leukocytes / physiology*
  • Leukocytosis / pathology
  • Leukocytosis / physiopathology
  • Mice
  • Mice, Inbred C57BL
  • Mice, Mutant Strains*
  • Myeloproliferative Disorders / pathology
  • Myeloproliferative Disorders / physiopathology*
  • Myxovirus Resistance Proteins
  • Neurofibromin 1 / genetics*
  • Signal Transduction / immunology
  • Splenomegaly / pathology
  • Splenomegaly / physiopathology
  • Viral Proteins / genetics
  • ras Proteins / metabolism

Substances

  • Mx1 protein, mouse
  • Myxovirus Resistance Proteins
  • Neurofibromin 1
  • Viral Proteins
  • Cre recombinase
  • Integrases
  • GTP-Binding Proteins
  • ras Proteins