Functional characterization of c-Mpl ectodomain mutations that underlie congenital amegakaryocytic thrombocytopenia

Growth Factors. 2014 Feb;32(1):18-26. doi: 10.3109/08977194.2013.874347. Epub 2014 Jan 20.

Abstract

Activation of the cell surface receptor, c-Mpl, by the cytokine, thrombopoietin (TPO), underpins megakaryocyte and platelet production in mammals. In humans, mutations in c-Mpl have been identified as the molecular basis of Congenital Amegakaryocytic Thrombocytopenia (CAMT). Here, we show that CAMT-associated mutations in c-Mpl principally lead to defective receptor presentation on the cell surface. In contrast, one CAMT mutant c-Mpl, F104S, was expressed on the cell surface, but showed defective TPO binding and receptor activation. Using mutational analyses, we examined which residues adjacent to F104 within the membrane-distal cytokine receptor homology module (CRM) of c-Mpl comprise the TPO-binding epitope, revealing residues within the predicted Domain 1 E-F and A-B loops and Domain 2 F'-G' loop as key TPO-binding determinants. These studies underscore the importance of the c-Mpl membrane-distal CRM to TPO-binding and suggest that mutations within this CRM that perturb TPO binding could give rise to CAMT.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Amino Acid Sequence
  • Animals
  • Binding Sites / genetics
  • Blood Platelets / metabolism
  • COS Cells
  • Cell Line
  • Cell Proliferation
  • Chlorocebus aethiops
  • Congenital Bone Marrow Failure Syndromes
  • Megakaryocytes / metabolism
  • Mice
  • Molecular Sequence Data
  • Mutation / genetics*
  • Oncogene Proteins / genetics
  • Protein Binding / genetics
  • Protein Structure, Tertiary / genetics
  • Receptors, Prolactin / genetics
  • Receptors, Thrombopoietin / genetics*
  • Sequence Alignment
  • Signal Transduction / genetics
  • Thrombocytopenia / genetics*
  • Thrombopoietin / metabolism*

Substances

  • Mpl protein, mouse
  • Oncogene Proteins
  • Receptors, Prolactin
  • Receptors, Thrombopoietin
  • Thrombopoietin

Supplementary concepts

  • Congenital amegakaryocytic thrombocytopenia