In vivo studies of mutant fibrillin-1 microfibrils

J Biol Chem. 2010 Aug 6;285(32):24943-55. doi: 10.1074/jbc.M110.130021. Epub 2010 Jun 7.

Abstract

In humans, mutations in fibrillin-1 result in a variety of genetic disorders with distinct clinical phenotypes. While most of the known mutations in fibrillin-1 cause Marfan syndrome, a number of other mutations lead to clinical features unrelated to Marfan syndrome. Pathogenesis of Marfan syndrome is currently thought to be driven by mechanisms due to haploinsufficiency of wild-type fibrillin-1. However, haploinsufficiency-driven mechanisms cannot explain the distinct phenotypes found in other fibrillinopathies. To test the hypothesis that mutations in fibrillin-1 cause disorders through primary effects on microfibril structure, two different mutations were generated in Fbn1 in mice. One mutation leads to a truncated fibrillin-1 molecule that is tagged with green fluorescent protein, allowing visualization of mutant fibrillin-1 incorporated into microfibrils. In heterozygosity, these mutant mice demonstrate progressive fragmentation of the aortic elastic lamellae and also display fragmentation of microfibrils in other tissues. Fibrillin-2 epitopes are also progressively revealed in these mice, suggesting that fibrillin-2 immunoreactivity can serve as a marker for microfibril degradation. In contrast, a second mutation (in-frame deletion of the first hybrid domain) in fibrillin-1 results in stable microfibrils, demonstrating that fibrillin-1 molecules are not required to be in perfect register for microfibril structure and function and that the first hybrid domain is dispensable for microfibril assembly. Taken together, these results suggest that perturbation of microfibril structure may underlie one of the major features of the Marfan syndrome: fragmentation of aortic elastic lamellae.

Publication types

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

MeSH terms

  • Alleles
  • Animals
  • Extracellular Matrix / metabolism
  • Fibrillin-1
  • Fibrillin-2
  • Fibrillins
  • Gene Deletion
  • Genotype
  • Humans
  • Marfan Syndrome / genetics
  • Mice
  • Mice, Transgenic
  • Microfibrils / metabolism*
  • Microfilament Proteins / chemistry
  • Microfilament Proteins / genetics*
  • Microscopy, Electron / methods
  • Models, Genetic
  • Mutation*

Substances

  • FBN1 protein, human
  • FBN2 protein, human
  • Fbn1 protein, mouse
  • Fbn2 protein, mouse
  • Fibrillin-1
  • Fibrillin-2
  • Fibrillins
  • Microfilament Proteins