L1077P CFTR pathogenic variant function rescue by Elexacaftor –Tezacaftor–Ivacaftor in cystic fibrosis patient-derived air–liquid interface (ALI) cultures and organoids: in vitro guided personalized therapy of non-F508del patients

Cystic fibrosis (CF) is caused by defects of the cystic fibrosis transmembrane conductance regulator (CFTR) gene. CFTR-modulating drugs may overcome specific defects, such as the case of Trikafta, which is a c...

Source: Respiratory Research - September 6, 2023 Category: Respiratory Medicine Authors: Lo Cicero Stefania, Castelli Germana, Blacon à Giovanna, Bruno Sabina Maria, Sette Giovanni, Pigliucci Riccardo, Villella Valeria Rachela, Esposito Speranza, Zollo Immacolata, Spadaro Francesca, De Maria Ruggero, Biffoni Mauro, Cimino Giuseppe, Amato Fel Tags: Research Source Type: research