In Utero and Postnatal Ivacaftor/Lumacaftor Therapy Rescues Multiorgan Disease in CFTR-F508del Ferrets

Using primary intestinal organoids and air-liquid interface cultures of airway epithelia, scientists demonstrated that the F508del mutation in ferret CF transmembrane conductance regulator (CFTR) results in a severe folding and trafficking defect, which could be partially restored by treatment with CFTR modulators.