Compound-heterozygous Marfan syndrome

Eur J Med Genet. 2009 Jan-Feb;52(1):1-5. doi: 10.1016/j.ejmg.2008.11.004. Epub 2008 Nov 27.

Abstract

We report two families in which the probands have compound-heterozygous Marfan syndrome (MFS). The proband of family 1 has the R2726W FBN1 mutation associated with isolated skeletal features on one allele and a pathogenic FBN1 mutation on the other allele. The phenotype of the compound-heterozygous probands appears to be more severe than that of their heterozygous family members which underlines the possibility that certain trans-located FBN1 mutations might act as modifiers of phenotype explaining some of the intrafamilial variability in Marfan syndrome.

Publication types

  • Case Reports

MeSH terms

  • Alleles
  • Family Health
  • Female
  • Fibrillin-1
  • Fibrillins
  • Heterozygote*
  • Humans
  • Male
  • Marfan Syndrome / genetics*
  • Microfilament Proteins / genetics*
  • Musculoskeletal Abnormalities / genetics
  • Mutation, Missense
  • Pedigree
  • Phenotype
  • Young Adult

Substances

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