Human disease-causing NOG missense mutations: effects on noggin secretion, dimer formation, and bone morphogenetic protein binding

Proc Natl Acad Sci U S A. 2001 Sep 25;98(20):11353-8. doi: 10.1073/pnas.201367598. Epub 2001 Sep 18.

Abstract

Secreted noggin protein regulates bone morphogenetic protein activity during development. In mice, a complete loss of noggin protein leads to multiple malformations including joint fusion, whereas mice heterozygous for Nog loss-of-function mutations are normal. In humans, heterozygous NOG missense mutations have been found in patients with two autosomal dominant disorders of joint development, multiple synostosis syndrome (SYNS1) and a milder disorder proximal symphalangism (SYM1). This study investigated the effect of one SYNS1 and two SYM1 disease-causing missense mutations on the structure and function of noggin. The SYNS1 mutation abolished, and the SYM1 mutations reduced, the secretion of functional noggin dimers in transiently transfected COS-7 cells. Coexpression of mutant noggin with wild-type noggin, to resemble the heterozygous state, did not interfere with wild-type noggin secretion. These data indicate that the human disease-causing mutations are hypomorphic alleles that reduce secretion of functional dimeric noggin. Therefore, we conclude that noggin has both species-specific and joint-specific dosage-dependent roles during joint formation. Surprisingly, in contrast to the COS-7 cell studies, the SYNS1 mutant was able to form dimers in Xenopus laevis oocytes. This finding indicates that there also exist species-specific differences in the ability to process mutant noggin polypeptides.

Publication types

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

MeSH terms

  • Animals
  • Bone Morphogenetic Proteins / antagonists & inhibitors
  • Bone Morphogenetic Proteins / metabolism*
  • COS Cells
  • Carrier Proteins
  • Chlorocebus aethiops
  • Dimerization
  • Disulfides
  • Female
  • Humans
  • Mutation, Missense*
  • Oocytes / physiology
  • Protein Biosynthesis
  • Proteins / genetics*
  • Proteins / metabolism*
  • Recombinant Proteins / metabolism
  • Synostosis / genetics
  • Transfection
  • Xenopus laevis

Substances

  • Bone Morphogenetic Proteins
  • Carrier Proteins
  • Disulfides
  • Proteins
  • Recombinant Proteins
  • noggin protein