How useful are cystic fibrosis mouse models?

Cystic fibrosis is a multi-system lethal genetic disorder. Mutations in the gene that encodes for the cystic fibrosis transmembrane conductance regulator (CFTR) are the basis of this disorder. Currently, a variety of CF mouse models have been developed for in-depth studies. They provide significant insight into the pathophysiology; however, these models do not always mimic the human disease. In this review we will summarize the available models and highlight the characteristics that have made them important tools for advancing our understanding of CF pathophysiology and for drug development.

Section editor:Gabriel Haddad – Pediatrics Department, University of California, San Diego, CA, USA