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

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

Abstract 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 clinically approved triple combination of Elexacaftor, Tezacaftor and Ivacaftor (ET...

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Main Authors: Stefania Lo Cicero, Germana Castelli, Giovanna Blaconà, Sabina Maria Bruno, Giovanni Sette, Riccardo Pigliucci, Valeria Rachela Villella, Speranza Esposito, Immacolata Zollo, Francesca Spadaro, Ruggero De Maria, Mauro Biffoni, Giuseppe Cimino, Felice Amato, Marco Lucarelli, Adriana Eramo
Format: Article
Language:English
Published: BMC 2023-09-01
Series:Respiratory Research
Subjects:
Cystic fibrosis
CFTR
Reprogrammed nasal cells
Nasal organoids
ALI culture
Trikafta
Online Access:https://doi.org/10.1186/s12931-023-02516-0

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