Development of a porcine model of cystic fibrosis

Trans Am Clin Climatol Assoc. 2009:120:149-62.

Abstract

Cystic Fibrosis (CF) is a common autosomal recessive disease that affects multiple organs. The lack of an animal model with manifestations like those typically found in humans has slowed understanding of its pathogenesis. Therefore, because of the similarities between human and swine anatomy, biochemistry, physiology, size, and genetics, we chose to develop a porcine model of CF. We used homologous recombination in primary cultures of porcine fibroblasts to disrupt the CFTR gene and then used those cells as nuclear donors for somatic cell nuclear transfer. After crossing heterozygous pigs, we produced CFTR-/- pigs. The newborn CFTR null piglets manifested meconium ileus, pancreatic destruction, early focal biliary cirrhosis, and gall bladder abnormalities that were very similar to those observed in humans with CF. At birth, there were no abnormalities in the airway epithelium or submucosal glands and no evidence of inflammation, consistent with findings in the newborn human. We hope that this porcine model will help elucidate the pathogenesis of CF and thereby lead to the development of new mechanism-based therapies.

Publication types

  • Comparative Study

MeSH terms

  • Animals
  • Animals, Genetically Modified
  • Animals, Newborn
  • Cystic Fibrosis / etiology
  • Cystic Fibrosis / genetics*
  • Cystic Fibrosis / metabolism
  • Cystic Fibrosis / pathology
  • Cystic Fibrosis Transmembrane Conductance Regulator / deficiency
  • Cystic Fibrosis Transmembrane Conductance Regulator / genetics
  • Disease Models, Animal
  • Gallbladder Diseases / etiology
  • Gallbladder Diseases / genetics
  • Gallbladder Diseases / pathology
  • Gene Targeting / methods
  • Humans
  • Ileus / etiology
  • Ileus / genetics
  • Infant, Newborn
  • Liver Diseases / etiology
  • Liver Diseases / genetics
  • Liver Diseases / pathology
  • Meconium
  • Models, Biological
  • Mutation
  • Pancreatic Diseases / etiology
  • Pancreatic Diseases / genetics
  • Pancreatic Diseases / pathology
  • Phenotype
  • Respiratory System / metabolism
  • Respiratory System / pathology
  • Species Specificity
  • Swine

Substances

  • CFTR protein, human
  • Cystic Fibrosis Transmembrane Conductance Regulator