Animal models of cystic fibrosis in the era of highly effective modulator therapies
BR Grubb, A Livraghi-Butrico - Current opinion in pharmacology, 2022 - Elsevier
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 alternatives for non-responders,
and the need to understand the kinetics of disease upon early life/chronic treatment. These …
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 alternatives for non-responders,
and the need to understand the kinetics of disease upon early life/chronic treatment. These …
