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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Autori principali:	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
Natura:	Artigo
Lingua:	Inglês
Pubblicazione:	BMC 2023-09-01
Serie:	Respiratory Research
Soggetti:	Cystic fibrosis CFTR Reprogrammed nasal cells Nasal organoids ALI culture Trikafta
Accesso online:	https://doi.org/10.1186/s12931-023-02516-0
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