Multiple congenital malformations of Wolf-Hirschhorn syndrome are recapitulated in Fgfrl1 null mice

Dis Model Mech. 2009 May-Jun;2(5-6):283-94. doi: 10.1242/dmm.002287. Epub 2009 Apr 21.

Abstract

Wolf-Hirschhorn syndrome (WHS) is caused by deletions in the short arm of chromosome 4 (4p) and occurs in about one per 20,000 births. Patients with WHS display a set of highly variable characteristics including craniofacial dysgenesis, mental retardation, speech problems, congenital heart defects, short stature and a variety of skeletal anomalies. Analysis of patients with 4p deletions has identified two WHS critical regions (WHSCRs); however, deletions targeting mouse WHSCRs do not recapitulate the classical WHS defects, and the genes contributing to WHS have not been conclusively established. Recently, the human FGFRL1 gene, encoding a putative fibroblast growth factor (FGF) decoy receptor, has been implicated in the craniofacial phenotype of a WHS patient. Here, we report that targeted deletion of the mouse Fgfrl1 gene recapitulates a broad array of WHS phenotypes, including abnormal craniofacial development, axial and appendicular skeletal anomalies, and congenital heart defects. Fgfrl1 null mutants also display a transient foetal anaemia and a fully penetrant diaphragm defect, causing prenatal and perinatal lethality. Together, these data support a wider role for Fgfrl1 in development, implicate FGFRL1 insufficiency in WHS, and provide a novel animal model to dissect the complex aetiology of this human disease.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Alleles
  • Anemia / complications
  • Animals
  • Animals, Newborn
  • Bone and Bones / abnormalities
  • Bone and Bones / pathology
  • Embryo, Mammalian / abnormalities
  • Embryo, Mammalian / pathology
  • Female
  • Fetus / abnormalities
  • Fetus / pathology
  • Gene Expression Regulation, Developmental
  • Gene Targeting
  • Heart Defects, Congenital / complications
  • Heart Septum / embryology
  • Heart Valves / embryology
  • Homozygote
  • Mice
  • Mice, Knockout
  • Placenta / embryology
  • Receptor, Fibroblast Growth Factor, Type 5 / deficiency*
  • Receptor, Fibroblast Growth Factor, Type 5 / genetics
  • Receptor, Fibroblast Growth Factor, Type 5 / metabolism
  • Recombination, Genetic / genetics
  • Sequence Homology, Nucleic Acid
  • Sex Characteristics
  • Wolf-Hirschhorn Syndrome / complications
  • Wolf-Hirschhorn Syndrome / pathology*

Substances

  • Fgfrl1 protein, mouse
  • Receptor, Fibroblast Growth Factor, Type 5