Pathogenic FBN1 variants in familial thoracic aortic aneurysms and dissections

Clin Genet. 2016 Jun;89(6):719-23. doi: 10.1111/cge.12702. Epub 2016 Jan 20.

Abstract

Marfan syndrome (MFS) due to mutations in FBN1 is a known cause of thoracic aortic aneurysms and acute aortic dissections (TAAD) associated with pleiotropic manifestations. Genetic predisposition to TAAD can also be inherited in families in the absence of syndromic features, termed familial TAAD (FTAAD), and several causative genes have been identified to date. FBN1 mutations can also be identified in FTAAD families, but the frequency of these mutations has not been established. We performed exome sequencing of 183 FTAAD families and identified pathogenic FBN1 variants in five (2.7%) of these families. We also identified eight additional FBN1 rare variants that could not be unequivocally classified as disease-causing in six families. FBN1 sequencing should be considered in individuals with FTAAD even without significant systemic features of MFS.

Keywords: FBN1; Marfan syndrome; familial thoracic aortic aneurysm and dissection.

Publication types

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

MeSH terms

  • Adult
  • Aged
  • Aortic Aneurysm, Thoracic / genetics*
  • Aortic Aneurysm, Thoracic / pathology
  • Aortic Dissection / genetics*
  • Aortic Dissection / pathology
  • Exome / genetics
  • Family Health
  • Female
  • Fibrillin-1 / genetics*
  • Genetic Predisposition to Disease / genetics*
  • Humans
  • Male
  • Marfan Syndrome / genetics
  • Marfan Syndrome / pathology
  • Middle Aged
  • Mutation*
  • Pedigree
  • Sequence Analysis, DNA / methods

Substances

  • FBN1 protein, human
  • Fibrillin-1

Supplementary concepts

  • Aortic Aneurysm, Familial Thoracic 1