Gain-of-function mutations in granulocyte colony-stimulating factor receptor (CSF3R) reveal distinct mechanisms of CSF3R activation

J Biol Chem. 2018 May 11;293(19):7387-7396. doi: 10.1074/jbc.RA118.002417. Epub 2018 Mar 23.

Abstract

Granulocyte colony-stimulating factor (G-CSF or CSF3) and its receptor CSF3R regulate granulopoiesis, neutrophil function, and hematopoietic stem cell mobilization. Recent studies have uncovered an oncogenic role of mutations in the CSF3R gene in many hematologic malignancies. To find additional CSF3R mutations that give rise to cell transformation, we performed a cellular transformation assay in which murine interleukin 3 (IL-3)-dependent Ba/F3 cells were transduced with WT CSF3R plasmid and screened for spontaneous growth in the absence of IL-3. Any outgrowth clones were sequenced to identify CSF3R mutations with transformation capacity. We identified several novel mutations and determined that they transform cells via four distinct mechanisms: 1) cysteine- and disulfide bond-mediated dimerization (S581C); 2) polar, noncharged amino acid substitution at the transmembrane helix dimer interface at residue Thr-640; 3) increased internalization by a Glu-524 substitution that mimics a low G-CSF dose; and 4) hydrophobic amino acid substitutions in the membrane-proximal residues Thr-612, Thr-615, and Thr-618. Furthermore, the change in signaling activation was related to an altered CSF3R localization. We also found that CSF3R-induced STAT3 and ERK activations require CSF3R internalization, whereas STAT5 activation occurred at the cell surface. Cumulatively, we have expanded the regions of the CSF3R extracellular and transmembrane domains in which missense mutations exhibit leukemogenic capacity and have further elucidated the mechanistic underpinnings that underlie altered CSF3R expression, dimerization, and signaling activation.

Keywords: CSF3R; cell biology; cell signaling; cell surface receptor; endocytosis; mutagenesis; receptor expression; signal transduction; signaling transduction.

Publication types

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

MeSH terms

  • Amino Acid Substitution
  • Animals
  • Cell Line
  • Cell Transformation, Neoplastic
  • Cysteine / metabolism
  • Dimerization
  • Disulfides / metabolism
  • Endocytosis
  • Gain of Function Mutation*
  • Humans
  • Hydrophobic and Hydrophilic Interactions
  • Leukemia / genetics
  • Mice
  • Mutagenesis
  • Receptors, Colony-Stimulating Factor / chemistry
  • Receptors, Colony-Stimulating Factor / genetics*
  • Receptors, Colony-Stimulating Factor / metabolism*
  • STAT3 Transcription Factor / metabolism
  • Signal Transduction
  • Subcellular Fractions / metabolism
  • Threonine / chemistry
  • Threonine / metabolism

Substances

  • CSF3R protein, human
  • Disulfides
  • Receptors, Colony-Stimulating Factor
  • STAT3 Transcription Factor
  • Threonine
  • Cysteine