Article
Theratyping cystic fibrosis in vitro in ALI culture and organoid models generated from patient-derived nasal epithelial conditionally reprogrammed stem cells.
The European respiratory journal - 1 Dec 2021
Sette Giovanni, Lo Cicero Stefania, Blaconà Giovanna, Pierandrei Silvia, Bruno Sabina Maria, Salvati Valentina, Castelli Germana, Falchi Mario, Fabrizzi Benedetta, Cimino Giuseppe, De Maria Ruggero, Biffoni Mauro, Eramo Adriana, Lucarelli Marco
Abstract excerpt
QUESTION: Cystic fibrosis (CF) is due to pathogenic variants in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Recent improvements have enabled pharmacological therapy aiming at restoring mutated CFTR expression and function. CFTR "modulators" have revolutionised the CF therapeutic landscape, particularly the last approved, Trikafta. This drug combination is indicated by the United States...
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