Dexamethasone normalizes aberrant elastic fiber production and collagen 1 secretion by Loeys-Dietz syndrome fibroblasts: a possible treatment?

Eur J Hum Genet. 2011 Jun;19(6):624-33. doi: 10.1038/ejhg.2010.259. Epub 2011 Jan 26.

Abstract

Loeys-Dietz syndrome (LDS) is an autosomal dominant connective tissue disorder characterized by facial dysmorphism, cleft palate, dilation of the aortic arch, blood vessel tortuosity and a high risk of aortic dissection. It is caused by mutations in the transforming growth factor β-receptor 1 and 2 (TGFβ-R1 and TGFβ-R2) genes. Fibroblasts derived from 12 Loeys-Dietz syndrome patients, six with TGFB-R1 mutations and six with TGFB-R2 mutations, were analyzed using RT-PCR, biochemical assays, immunohistochemistry and electron microscopy for production of elastin, fibrillin 1, fibulin 1 and fibulin 4 and deposition of collagen type I. All LDS fibroblasts with TGFβ-R1 mutations demonstrated decreased expression of elastin and fibulin 1 genes and impaired deposition of elastic fibers. In contrast, fibroblasts with TGFβ-R2 mutations consistently demonstrated intracellular accumulation of collagen type I in the presence of otherwise normal elastic fiber production. Treatment of the cell cultures with dexamethasone induced remarkable upregulation in the expression of tropoelastin, fibulin 1- and fibulin 4-encoding mRNAs, leading to normalization of elastic fiber production in fibroblasts with TGFβ-R1 mutations. Treatment with dexamethasone also corrected the abnormal secretion of collagen type I from fibroblasts with TGFβ-R2 gene mutations. As the organogenesis-relevant elastic fiber production occurs exclusively in late fetal and early neonatal life, these findings may have implications for treatment in early life. Further studies are required to determine if dexamethasone treatment of fetuses prenatally diagnosed with LDS would prevent or alleviate the connective tissue and vascular defects seen in this syndrome.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Calcium-Binding Proteins / biosynthesis
  • Cell Culture Techniques
  • Collagen / biosynthesis*
  • Collagen / metabolism
  • Dexamethasone / pharmacology
  • Dexamethasone / therapeutic use*
  • Elastic Tissue / drug effects*
  • Elastic Tissue / metabolism
  • Elastic Tissue / pathology
  • Elastin / biosynthesis
  • Extracellular Matrix Proteins / biosynthesis
  • Female
  • Fetus
  • Fibrillin-1
  • Fibrillins
  • Fibroblasts / metabolism*
  • Fibroblasts / pathology
  • Genetic Testing
  • Humans
  • Immunohistochemistry
  • Loeys-Dietz Syndrome* / drug therapy
  • Loeys-Dietz Syndrome* / genetics
  • Loeys-Dietz Syndrome* / pathology
  • Microfilament Proteins / biosynthesis
  • Microscopy, Electron
  • Pregnancy
  • Protein Serine-Threonine Kinases / genetics*
  • Protein Serine-Threonine Kinases / metabolism
  • Receptor, Transforming Growth Factor-beta Type I
  • Receptor, Transforming Growth Factor-beta Type II
  • Receptors, Transforming Growth Factor beta / genetics*
  • Receptors, Transforming Growth Factor beta / metabolism
  • Reverse Transcriptase Polymerase Chain Reaction
  • Tropoelastin / biosynthesis

Substances

  • Calcium-Binding Proteins
  • EFEMP2 protein, human
  • Extracellular Matrix Proteins
  • FBN1 protein, human
  • Fibrillin-1
  • Fibrillins
  • Microfilament Proteins
  • Receptors, Transforming Growth Factor beta
  • Tropoelastin
  • fibulin
  • Dexamethasone
  • Collagen
  • Elastin
  • Protein Serine-Threonine Kinases
  • Receptor, Transforming Growth Factor-beta Type I
  • Receptor, Transforming Growth Factor-beta Type II