CFTR Mouse Models

Why Model CFTR

Cftr is the mouse ortholog of human CFTR and encodes the cystic fibrosis transmembrane conductance regulator, an ATP-binding-cassette protein that functions as a chloride and bicarbonate channel at epithelial surfaces. CFTR has been associated with regulation of fluid and ion transport.

Mutations in CFTR, including the common F508del, cause cystic fibrosis, and CFTR modulator therapies are in clinical use. Cftr-deficient and mutant mice reproduce some features of the disease, with differences from the human condition.

Model Applications

Humanized CFTR models, including those carrying human disease-associated alleles, have been used to study CFTR function and to evaluate CFTR-directed therapeutics in defined experimental settings.

Custom CFTR Model Options

Engineered to your specifications, with molecular validation of the targeted allele and germline transmission guaranteed.

• Conventional knockout
• Conditional knockout
• Point mutation / knockin
• Reporter knockin
• Humanized
• Targeted transgenic

Sources

• Unraveling the mechanism of action, binding sites, and therapeutic advances of CFTR modulators: a narrative review. Curr Issues Mol Biol. 2025. doi:10.3390/cimb47020119
• Riordan JR et al. Identification of the cystic fibrosis gene: cloning and characterization of complementary DNA. Science. 1989. doi:10.1126/science.2475911

Last reviewed: October 22, 2026 · Reviewed by: Paul Sheiffele

Related gene targets: Scnn1b · Slc26a9 · Ano1 · Muc5b