In Utero CFTR Modulation Alleviates Disease in G551D Cystic Fibrosis Pigs
Sarah E. Ernst, David Kyle Meyerholz, Melissa Samuel…·David Anthony Stoltz
This paper shows that in utero ivacaftor treatment rescues multiple organ defects in CFTR-G551D pigs, improving pancreatic, gallbladder, and vas deferens function.