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 clinically approved triple combination of Elexacaftor, Tezacaftor and Ivacaftor (ETI) that e...

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Detalles Bibliográficos
Autores principales: Lo Cicero, Stefania, Castelli, Germana, Blaconà, Giovanna, Bruno, Sabina Maria, Sette, Giovanni, Pigliucci, Riccardo, Villella, Valeria Rachela, Esposito, Speranza, Zollo, Immacolata, Spadaro, Francesca, Maria, Ruggero De, Biffoni, Mauro, Cimino, Giuseppe, Amato, Felice, Lucarelli, Marco, Eramo, Adriana
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2023
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10483775/
https://www.ncbi.nlm.nih.gov/pubmed/37674160
http://dx.doi.org/10.1186/s12931-023-02516-0

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10483775/
https://www.ncbi.nlm.nih.gov/pubmed/37674160
http://dx.doi.org/10.1186/s12931-023-02516-0

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