Intestinal Current Measurements Detect Activation of Mutant CFTR in Cystic Fibrosis Patients with the G551D Mutation Treated with Ivacaftor
S Gräber, M Hug, O Sommerburg, S Hirtz, J Hentschel, A Heinzmann, C Dopfer, A Schulz, J Mainz, B Tümmler, MA Mall · Pneumologie · 2015 · Heft 07
Bibliografische Angaben
Zusammenfassung
Introduction: Recent studies suggest that sensitive outcome measures of in vivo CFTR activity may be important to accelerate clinical development of emerging CFTR modulator therapies for cystic fibrosis (CF). Intestinal current measurements (ICM) detect pharmacological activation of mutant CFTR ex vivo. However, whether ICM can detect rescue of mutant CFTR function in CF patients remains unknown. Objective: To determine if ICM is sensitive to detect rescue of CFTR Cl- channel function in CF patients with the G551D-CFTR mutation after ivacaftor initiation. Methods: Rectal biopsies were obtained from 37 non-CF con…
