TGFBR1 mutations associated with Loeys-Dietz syndrome are inactivating

J Recept Signal Transduct Res. 2012 Jun;32(3):150-5. doi: 10.3109/10799893.2012.664553. Epub 2012 Mar 14.

Abstract

To assess the effect of Loeys-Dietz syndrome (LDS) mutations affecting TGFΒR1 a selection of seven disease-associated amino acid substitutions were introduced into wild type TGFβR1 and constitutively active TGFβR1(T204D). Receptor function was tested by co-transfection with a luciferase reporter or EGFP-tagged SMAD2 in HEK293 cells. All of the mutations were found to be inactivating for canonical TGF-β signaling. Differences in residual activity were not found to correlate with disease subtype. In co-transfection experiments with equal amounts wild-type receptor, the LDS mutations were found to confer a modest dominant negative effect. These results are discussed in relation to LDS and the related Marfan syndrome.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Aortic Aneurysm / genetics
  • Genes, Dominant
  • HEK293 Cells
  • Humans
  • Loeys-Dietz Syndrome / genetics
  • Marfan Syndrome / genetics
  • Mutation*
  • Phenotype
  • Phosphorylation
  • Protein Serine-Threonine Kinases / genetics*
  • Receptor, Transforming Growth Factor-beta Type I
  • Receptors, Transforming Growth Factor beta / genetics*
  • Signal Transduction / genetics
  • Smad Proteins / metabolism
  • Transfection
  • Transforming Growth Factor beta / genetics

Substances

  • Receptors, Transforming Growth Factor beta
  • Smad Proteins
  • Transforming Growth Factor beta
  • Protein Serine-Threonine Kinases
  • Receptor, Transforming Growth Factor-beta Type I
  • TGFBR1 protein, human