Identification of a new gene mutated in Fraser syndrome and mouse myelencephalic blebs

Nat Genet. 2005 May;37(5):520-5. doi: 10.1038/ng1549. Epub 2005 Apr 17.

Abstract

Fraser syndrome is a recessive, multisystem disorder presenting with cryptophthalmos, syndactyly and renal defects and associated with loss-of-function mutations of the extracellular matrix protein FRAS1. Fras1 mutant mice have a blebbed phenotype characterized by intrauterine epithelial fragility generating serous and, later, hemorrhagic blisters. The myelencephalic blebs (my) strain has a similar phenotype. We mapped my to Frem2, a gene related to Fras1 and Frem1, and showed that a Frem2 gene-trap mutation was allelic to my. Expression of Frem2 in adult kidneys correlated with cyst formation in my homozygotes, indicating that the gene is required for maintaining the differentiated state of renal epithelia. Two individuals with Fraser syndrome were homozygous with respect to the same missense mutation of FREM2, confirming genetic heterogeneity. This is the only missense mutation reported in any blebbing mutant or individual with Fraser syndrome, suggesting that calcium binding in the CALXbeta-cadherin motif is important for normal functioning of FREM2.

Publication types

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

MeSH terms

  • Animals
  • Blister / genetics*
  • Extracellular Matrix Proteins / genetics*
  • Eyelids / abnormalities
  • Genitalia / abnormalities
  • Humans
  • Medulla Oblongata / pathology*
  • Mice
  • Molecular Sequence Data
  • Syndactyly / genetics

Substances

  • Extracellular Matrix Proteins
  • FREM2 protein, human
  • Fras1 protein, mouse

Associated data

  • GENBANK/AJ833643
  • GENBANK/BN000687