Generation of disease-specific iPSCs and development of transgenic reporter cell lines for cystic fibrosis disease modelling and drug screening
M Schubert, S Merkert, A Haase, L Engels, R Haller, N Lachmann, T Moritz, B Tümmler, L Galietta, U Martin · Pneumologie · 2015 · Heft 07
Bibliografische Angaben
Zusammenfassung
Introduction: Cystic fibrosis (CF) is a genetic disorder caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene coding for a cAMP-activated chloride-channel expressed in different epithelia. So far, immortalized cell lines overexpressing mutant CFTR-variants have been used to screen compound libraries. In fact, CFTR-protein-modulators have been identified, but show modest effects at best. Obviously, the complexity of the mutant CFTR-maturation and turnover kinetics including the influence of genetic modifiers require the use of advanced personalized cellular models that closel…
