Disruption of the gene encoding the latent transforming growth factor-beta binding protein 4 (LTBP-4) causes abnormal lung development, cardiomyopathy, and colorectal cancer

Genes Dev. 2002 Sep 1;16(17):2264-73. doi: 10.1101/gad.229102.

Abstract

Transforming growth factor-betas (TGF-betas) are multifunctional growth factors that are secreted as inactive (latent) precursors in large protein complexes. These complexes include the latency-associated propeptide (LAP) and a latent transforming growth factor-beta binding protein (LTBP). Four isoforms of LTBPs (LTBP-1-LTBP-4) have been cloned and are believed to be structural components of connective tissue microfibrils and local regulators of TGF-beta tissue deposition and signaling. By using a gene trap strategy that selects for integrations into genes induced transiently during early mouse development, we have disrupted the mouse homolog of the human LTBP-4 gene. Mice homozygous for the disrupted allele develop severe pulmonary emphysema, cardiomyopathy, and colorectal cancer. These highly tissue-specific abnormalities are associated with profound defects in the elastic fiber structure and with a reduced deposition of TGF-beta in the extracellular space. As a consequence, epithelial cells have reduced levels of phosphorylated Smad2 proteins, overexpress c-myc, and undergo uncontrolled proliferation. This phenotype supports the predicted dual role of LTBP-4 as a structural component of the extracellular matrix and as a local regulator of TGF-beta tissue deposition and signaling.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adaptor Proteins, Signal Transducing*
  • Animals
  • Cardiomyopathies / genetics*
  • Cardiomyopathies / metabolism
  • Cardiomyopathies / pathology
  • Carrier Proteins / genetics*
  • Carrier Proteins / metabolism
  • Colorectal Neoplasms / genetics*
  • Colorectal Neoplasms / metabolism
  • Colorectal Neoplasms / pathology
  • Elastic Tissue / metabolism
  • Elastic Tissue / pathology
  • Extracellular Matrix / metabolism
  • Gene Expression Regulation, Developmental
  • Gene Targeting
  • Humans
  • Introns
  • Latent TGF-beta Binding Proteins / genetics*
  • Latent TGF-beta Binding Proteins / metabolism
  • Lung / abnormalities*
  • Lung / metabolism
  • Mice
  • Mice, Inbred C57BL
  • Mice, Knockout
  • Mice, Transgenic
  • Phenotype
  • Pulmonary Emphysema / genetics
  • Pulmonary Emphysema / metabolism
  • Pulmonary Emphysema / pathology
  • Signal Transduction
  • Transforming Growth Factor beta / metabolism
  • Transforming Growth Factor beta1

Substances

  • Adaptor Proteins, Signal Transducing
  • Carrier Proteins
  • LTBP-4 protein, mouse
  • LTBP1 protein, human
  • LTBP4 protein, human
  • Latent TGF-beta Binding Proteins
  • Ltbp1 protein, mouse
  • TGFB1 protein, human
  • Tgfb1 protein, mouse
  • Transforming Growth Factor beta
  • Transforming Growth Factor beta1