Correction of CFTR function in intestinal organoids to guide treatment of cystic fibrosis

• Published in: The European respiratory journal Vol. 57; no. 1; p. 1902426
• Main Authors: Ramalho, Anabela S., Fürstová, Eva, Vonk, Annelotte M., Ferrante, Marc, Verfaillie, Catherine, Dupont, Lieven, Boon, Mieke, Proesmans, Marijke, Beekman, Jeffrey M., Sarouk, Ifat, Vazquez Cordero, Carlos, Vermeulen, Francois, De Boeck, Kris
• Format: Journal Article
• Language: English
• Published: England 01.01.2021

Given the vast number of cystic fibrosis transmembrane conductance regulator ( ) mutations, biomarkers predicting benefit from CFTR modulator therapies are needed for subjects with cystic fibrosis (CF). To study CFTR function in organoids of subjects with common and rare mutations and evaluate corre...