Characteristic multiorgan pathology of cystic fibrosis in a long-living cystic fibrosis transmembrane regulator knockout murine model

Am J Pathol. 2004 Apr;164(4):1481-93. doi: 10.1016/S0002-9440(10)63234-8.

Abstract

The lack of an appropriate animal model with multiorgan pathology characteristic of the human form of cystic fibrosis has hampered our understanding of the pathobiology of the disease. We evaluated multiple organs of congenic C57BL/6J cystic fibrosis transmembrane regulator (Cftr)(-/-) and Cftr(+/+) mice maintained from weaning on a liquid diet then sacrificed between 1 and 24 months of age. The lungs of the Cftr(-/-) animals showed patchy alveolar overdistention, interstitial thickening, and fibrosis, with progression up to 6 months of age. The proximal and distal airway surface was encased with mucus-like material but lacked overt evidence of chronic bacterial infections or inflammation. All Cftr(-/-) animals showed progressive liver disease, with hepatosteatosis, focal cholangitis, inspissated secretions, and bile duct proliferation; after 1 year of age there was progression to focal biliary cirrhosis. The intercalated, intralobular and interlobular ducts and acinar lumina of the exocrine pancreas, the parotid and submaxillary glands of the Cftr(-/-) animals were dilated and filled with inspissated material, as well as mild inflammation and acinar cell drop out. Quantitative measurements of the pancreas showed significant acinar atrophy and increased acinar volume in comparison with age-matched Cftr(+/+) littermates. The ileal lumen and crypts were filled with adherent fibrillar material. After 3 months of age the vas deferens of the Cftr(-/-) animals could not be identified. None of the aforementioned pathological changes were observed in the Cftr(+/+) littermates fed the same liquid diet. We show, for the first time, that long-lived C578L/6J Cftr(-/-) mice develop manifestations of cystic fibrosis-like disease in all pathologically affected organs in the human form of cystic fibrosis.

Publication types

  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Animals
  • Cystic Fibrosis / pathology*
  • Cystic Fibrosis Transmembrane Conductance Regulator / genetics*
  • Diet
  • Disease Models, Animal*
  • Female
  • Intestines / pathology
  • Intestines / ultrastructure
  • Liver / pathology
  • Liver / ultrastructure
  • Lung / pathology
  • Lung / ultrastructure
  • Male
  • Mice
  • Mice, Congenic
  • Mice, Inbred CFTR
  • Mice, Knockout
  • Microscopy, Electron
  • Pancreas / pathology
  • Pancreas / ultrastructure
  • Salivary Glands / pathology
  • Salivary Glands / ultrastructure
  • Time Factors
  • Vas Deferens / pathology
  • Vas Deferens / ultrastructure

Substances

  • Cystic Fibrosis Transmembrane Conductance Regulator