Article
Correction of CFTR function in intestinal organoids to guide treatment of cystic fibrosis.
The European respiratory journal - 1 Jan 2021
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
Abstract excerpt
RATIONALE: Given the vast number of cystic fibrosis transmembrane conductance regulator (CFTR) mutations, biomarkers predicting benefit from CFTR modulator therapies are needed for subjects with cystic fibrosis (CF). OBJECTIVES: To study CFTR function in organoids of subjects with common and rare CFTR mutations and evaluate correlations between CFTR function and clinical data. METHODS: Intestinal organoids were...
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