Ferret and pig models of cystic fibrosis: prospects and promise for gene therapy

Hum Gene Ther Clin Dev. 2015 Mar;26(1):38-49. doi: 10.1089/humc.2014.154. Epub 2015 Feb 12.


Large animal models of genetic diseases are rapidly becoming integral to biomedical research as technologies to manipulate the mammalian genome improve. The creation of cystic fibrosis (CF) ferrets and pigs is an example of such progress in animal modeling, with the disease phenotypes in the ferret and pig models more reflective of human CF disease than mouse models. The ferret and pig CF models also provide unique opportunities to develop and assess the effectiveness of gene and cell therapies to treat affected organs. In this review, we examine the organ disease phenotypes in these new CF models and the opportunities to test gene therapies at various stages of disease progression in affected organs. We then discuss the progress in developing recombinant replication-defective adenoviral, adeno-associated viral, and lentiviral vectors to target genes to the lung and pancreas in ferrets and pigs, the two most affected organs in CF. Through this review, we hope to convey the potential of these new animal models for developing CF gene and cell therapies.

Publication types

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

MeSH terms

  • Animals
  • Cystic Fibrosis / therapy*
  • Disease Models, Animal*
  • Ferrets*
  • Genetic Therapy*
  • Genetic Vectors
  • Lung / metabolism
  • Pancreas / metabolism
  • Swine*