A novel FBN1 variant in a large Marfan family with high penetrance of aortic dissection or rupture

Dan Med J. 2014 Nov;61(11):A4949.

Abstract

Introduction: Marfan syndrome is an autosomal, dominantly inherited disorder of the connective tissue. We report the clinical data and results of a genetic analysis of a large Danish Marfan family.

Methods: Sanger sequencing of FBN1 was initially performed on genomic DNA from the index patient. Subsequently, four affected family members and three non-affected family members were tested for the variant identified in the index patient.

Results: A novel variant (c.701G>T) in the FBN1 segregated with Marfan features in the family.

Conclusion: In the majority of the family members, this novel variant seems to cause a uniform and very detrimental set of disease characteristics including fatal aortic dissection.

Funding: not relevant.

Trial registration: not relevant.

Publication types

  • Case Reports

MeSH terms

  • Aortic Rupture / genetics
  • Denmark / ethnology
  • Exons / genetics
  • Fibrillin-1
  • Fibrillins
  • Genetic Association Studies
  • Genetic Variation
  • Humans
  • Marfan Syndrome / genetics*
  • Microfilament Proteins / genetics*
  • Pedigree
  • Penetrance
  • Retrospective Studies

Substances

  • FBN1 protein, human
  • Fibrillin-1
  • Fibrillins
  • Microfilament Proteins