Fibulin-5 mutations: mechanisms of impaired elastic fiber formation in recessive cutis laxa

Hum Mol Genet. 2006 Dec 1;15(23):3379-86. doi: 10.1093/hmg/ddl414. Epub 2006 Oct 11.

Abstract

To elucidate the molecular mechanisms of impaired elastic fiber formation in recessive cutis laxa, we have investigated two disease-causing missense substitutions in fibulin-5, C217R and S227P. Pulse-chase immunoprecipitation experiments indicated that S227P mutant fibulin-5 was synthesized and secreted by skin fibroblasts at a reduced rate when compared with the wild-type protein. Both mutants failed to be incorporated into elastic fibers by transfected rat lung fibroblasts. Purified recombinant fibulin-5 with either mutation showed reduced affinity for tropoelastin in solid-phase binding assays. Furthermore, S227P mutant fibulin-5 also showed impaired association with fibrillin-1 microfibrils. The same mutation triggered an endoplasmic reticulum (ER) stress response, as indicated by the strong co-localization of this mutant protein with folding chaperones in the ER, including calreticulin, immunoglobulin-binding protein and protein disulfide isomerase, and by increased rates of apoptosis in patient fibroblasts. Histological analysis of skin sections from a cutis laxa patient with a homozygous S227P mutation showed a lack of fibulin-5 in the extracellular matrix and a concomitant disorganization of dermal elastic fibers. By electron microscopy, elastic fibers in the skin of this patient showed a failure of elastin globules to fuse into a continuous elastic fiber core. We conclude that recessive cutis laxa mutations in fibulin-5 result in misfolding, decreased secretion and a reduced interaction with elastin and fibrillin-1 leading to impaired elastic fiber development. These findings support the hypothesis that fibulin-5 is necessary for elastic fiber formation by facilitating the deposition of elastin onto a microfibrillar scaffold via direct molecular interactions.

Publication types

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

MeSH terms

  • Amino Acid Substitution
  • Animals
  • Calreticulin / analysis
  • Calreticulin / metabolism
  • Cutis Laxa / genetics*
  • Cutis Laxa / metabolism
  • Cutis Laxa / pathology*
  • Elastic Tissue / growth & development
  • Elastic Tissue / metabolism*
  • Elastic Tissue / ultrastructure*
  • Endoplasmic Reticulum / chemistry
  • Endoplasmic Reticulum / metabolism
  • Extracellular Matrix Proteins / analysis
  • Extracellular Matrix Proteins / genetics*
  • Extracellular Matrix Proteins / metabolism
  • Fibrillin-1
  • Fibrillins
  • Fibroblasts / metabolism
  • Fibroblasts / pathology
  • Genes, Recessive
  • Humans
  • Immunoprecipitation
  • Microfilament Proteins / analysis
  • Microfilament Proteins / metabolism
  • Mutation, Missense
  • Prostatic Secretory Proteins / metabolism
  • Protein Disulfide-Isomerases / metabolism
  • Protein Folding
  • Rats
  • Skin / metabolism
  • Skin / pathology*
  • Tropoelastin / metabolism

Substances

  • Calreticulin
  • Extracellular Matrix Proteins
  • FBLN5 protein, human
  • FBN1 protein, human
  • Fbn1 protein, rat
  • Fibrillin-1
  • Fibrillins
  • Microfilament Proteins
  • Prostatic Secretory Proteins
  • Tropoelastin
  • beta-microseminoprotein
  • Protein Disulfide-Isomerases