Animal models of cystic fibrosis in the era of highly effective modulator therapies.

Abstract excerpt

Few human genetic diseases can rely on the availability of as many and as diverse animal models as cystic fibrosis (CF), a multiorgan syndrome caused by functional absence of cystic fibrosis transmembrane regulator (CFTR). The recent development of highly effective CFTR modulator drug therapies simultaneously highlighted the remarkable clinical improvement achievable with these treatments, the lack of therapeutic...

Topics

Join the communities discussing this publication.

• Cystic Fibrosis
• Cystic Fibrosis Transmembrane Conductance Regulator
• Models, Animal
• Mutation