Endoglin, a TGF-beta binding protein of endothelial cells, is the gene for hereditary haemorrhagic telangiectasia type 1

Nat Genet. 1994 Dec;8(4):345-51. doi: 10.1038/ng1294-345.

Abstract

Hereditary haemorrhagic telangiectasia (HHT) is an autosomal dominant disorder characterized by multisystemic vascular dysplasia and recurrent haemorrhage. Linkage for some families has been established to chromosome 9q33-q34. In the present study, endoglin, a transforming growth factor beta (TGF-beta) binding protein, was analysed as a candidate gene for the disorder based on chromosomal location, expression pattern and function. We have identified mutations in three affected individuals: a C to G substitution converting a tyrosine to a termination codon, a 39 base pair deletion and a 2 basepair deletion which creates a premature termination codon. We have identified endoglin as the HHT gene mapping to 9q3 and have established HHT as the first human disease defined by a mutation in a member of the TGF-beta receptor complex.

Publication types

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

MeSH terms

  • Amino Acid Sequence
  • Antigens, CD
  • Base Sequence
  • Chromosome Mapping
  • Chromosomes, Human, Pair 9
  • Codon
  • DNA, Complementary
  • Endoglin
  • Female
  • Humans
  • Male
  • Membrane Glycoproteins / genetics*
  • Membrane Glycoproteins / metabolism
  • Molecular Sequence Data
  • Pedigree
  • Receptors, Cell Surface
  • Telangiectasia, Hereditary Hemorrhagic / genetics*
  • Terminator Regions, Genetic
  • Transforming Growth Factor beta / metabolism*
  • Vascular Cell Adhesion Molecule-1*

Substances

  • Antigens, CD
  • Codon
  • DNA, Complementary
  • ENG protein, human
  • Endoglin
  • Membrane Glycoproteins
  • Receptors, Cell Surface
  • Transforming Growth Factor beta
  • Vascular Cell Adhesion Molecule-1