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Scientific research article presenting a Belgian multicenter study comparing drug responses in patient-derived intestinal organoids with clinical outcomes after CFTR modulator therapy in individuals with cystic fibrosis homozygous for the F508del mutation.
Authors: Noelia Rodriguez Mier; Senne Cuyx; Kris De Boeck; Marijke Proesmans; Mieke Boon; Lieven Dupont; Kaline Arnauts; Elke De Wachter; Stephanie Van Biervliet; Monique Lequesne; Vicky Nowé; Hedwige Boboli; Anabela Santo Ramalho; François Vermeulen; on behalf of the Belgian Organoid Project
Affiliations: KU Leuven; University Hospital Leuven; University Hospital Brussels; Ghent University Hospital; Antwerp University Hospital; GZA Hospital; CHR Citadelle
Country: Belgium
Research field: Cystic fibrosis; CFTR modulators; organoid research; personalized medicine
Study population: 60 people with cystic fibrosis homozygous for F508del
Genetic focus: F508del CFTR mutation (homozygous)
Interventions studied: Elexacaftor/tezacaftor/ivacaftor; Tezacaftor/ivacaftor
Methods: Patient-derived intestinal organoids from rectal biopsies; forskolin-induced swelling assay; comparison with clinical outcomes
Clinical outcomes assessed: Lung function; sweat chloride levels; body weight; pulmonary exacerbations
Type of document: Scientific research article
Source: PubMed
DOI / Identifier: 40883119
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