Novel exon nucleotide substitution at the splice junction causes a neonatal Marfan syndrome

Clin Genet. 2010 May;77(5):453-63. doi: 10.1111/j.1399-0004.2009.01337.x. Epub 2010 Feb 4.

Abstract

The fibrillin-1 gene (FBN1) mutations are associated with a broad spectrum of disorders including Marfan syndrome (MFS) and show great clinical heterogeneity. An underrepresentation for mutations leading to premature termination codon (PTC) in FBN1 exons 24-32 was found in neonatal or severe MFS but the underlying cause was unclear. This study thoroughly examined two FBN1 mutations on exons 24-32 region to illustrate the molecular mechanisms underlying these FBN1 mutations on MFS etiology. Two nucleotide substitutions, c.3208G> C, the last nucleotide of exon 26, and c.3209A>G, the first nucleotide of exon 27, affecting the same amino acid, p.D1070H and p.D1070G, respectively, gave very different phenotypes. We demonstrate that c.3208G>C generates two alternatively spliced transcripts, while c.3209A>G does not affect the splicing. We further demonstrate that the aberrantly spliced transcripts do not go through nonsense-mediated decay, but rather produce unstable, premature protein peptides that are degraded by endoplasmic reticulum associated degradation. The molecular mechanism outlined here defines a model for the pathogenesis of PTC-containing mutation within the exons 24-32 of FBN1 in MFS. Furthermore, our data suggest that PTC mutation within this region may lead to early lethality in neonatal MFS.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Base Sequence
  • Blotting, Western
  • Cell Line
  • Codon, Nonsense / genetics
  • Endoplasmic Reticulum / drug effects
  • Endoplasmic Reticulum / metabolism
  • Exons / genetics*
  • Fatal Outcome
  • Female
  • Fibrillin-1
  • Fibrillins
  • Humans
  • Infant, Newborn
  • Intracellular Space / drug effects
  • Intracellular Space / metabolism
  • Leupeptins / pharmacology
  • Marfan Syndrome / genetics*
  • Microfilament Proteins / genetics
  • Microscopy, Fluorescence
  • Molecular Sequence Data
  • Mutant Proteins / metabolism
  • Mutation / genetics
  • Nucleotides / genetics*
  • Pregnancy
  • Protein Processing, Post-Translational / drug effects
  • RNA Splice Sites / drug effects
  • RNA Splice Sites / genetics*
  • RNA Splicing / drug effects
  • RNA Splicing / genetics
  • RNA Stability / drug effects
  • RNA Stability / genetics

Substances

  • Codon, Nonsense
  • FBN1 protein, human
  • Fibrillin-1
  • Fibrillins
  • Leupeptins
  • Microfilament Proteins
  • Mutant Proteins
  • Nucleotides
  • RNA Splice Sites
  • benzyloxycarbonylleucyl-leucyl-leucine aldehyde