Mutation of the gene encoding fibrillin-2 results in syndactyly in mice

Hum Mol Genet. 2001 Apr 1;10(8):835-43. doi: 10.1093/hmg/10.8.835.

Abstract

Fibrillins are large, cysteine-rich glycoproteins that form microfibrils and play a central role in elastic fibrillogenesis. Fibrillin-1 and fibrillin-2, encoded by FBN1 on chromosome 15q21.1 and FBN2 on chromosome 5q23-q31, are highly similar proteins. The finding of mutations in FBN1 and FBN2 in the autosomal dominant microfibrillopathies Marfan syndrome (MFS) and congenital contractural arachnodactyly (CCA), respectively, has highlighted their essential role in the development and homeostasis of elastic fibres. MFS is characterized by cardiovascular, skeletal and ocular abnormalities, and CCA by long, thin, flexed digits, crumpled ears and mild joint contractures. Although mutations arise throughout FBN1, those clustering within exons 24-32 are associated with the most severe form of MFS, so-called neonatal MFS. All the mutations described in CCA occur in the "neonatal region" of FBN2. Both MFS and CCA are thought to arise via a dominant negative mechanism. The analysis of mouse mutations has demonstrated that fibrillin-1 microfibrils are mainly engaged in tissue homeostasis rather than elastic matrix assembly. In the current investigation, we have analysed the classical mouse mutant shaker-with-syndactylism using a positional candidate approach and demonstrated that loss-of-function mutations outside the "neonatal region" of Fbn2 cause syndactyly in mice. These results suggest that phenotypes distinct from CCA may result in man as a consequence of mutations outside the "neonatal region" of FBN2.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Amino Acid Sequence
  • Animals
  • Exons
  • Fibrillin-1
  • Fibrillin-2
  • Fibrillins
  • Glycoproteins / genetics*
  • Heterozygote
  • Mice
  • Mice, Inbred Strains
  • Microfilament Proteins / chemistry
  • Microfilament Proteins / genetics*
  • Molecular Sequence Data
  • Mutation*
  • Protein Structure, Secondary
  • Sequence Deletion
  • Syndactyly / genetics*
  • Transforming Growth Factor beta / metabolism

Substances

  • Fbn1 protein, mouse
  • Fbn2 protein, mouse
  • Fibrillin-1
  • Fibrillin-2
  • Fibrillins
  • Glycoproteins
  • Microfilament Proteins
  • Transforming Growth Factor beta