Elexacaftor/tezacaftor/ivacaftor efficacy in intestinal organoids with rare CFTR variants in comparison to CFTR-F508del and CFTR-wild type controls.

doi:10.1016/j.jcf.2024.09.019

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Article

Elexacaftor/tezacaftor/ivacaftor efficacy in intestinal organoids with rare CFTR variants in comparison to CFTR-F508del and CFTR-wild type controls.

Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society - 1 Jan 2025

Kroes Suzanne, Bierlaagh Marlou C, Lefferts Juliet W, Boni Alessandra, Muilwijk Danya, Viscomi Carla, Keijzer-Nieuwenhuijze Natascha D A, Cristiani Luca, Niemöller Paul J, Verburg Tibo F, Cutrera Renato, Fiocchi Alessandro G, Lucidi Vincenzina, van der Ent Cornelis K, Beekman Jeffrey M, Alghisi Federico, Ciciriello Fabiana

Abstract excerpt

Cystic fibrosis is a life-shortening genetic disease caused by pathological variants of the cystic fibrosis transmembrane conductance regulator gene. The CFTR modulator therapy elexacaftor, tezacaftor and ivacaftor (ETI) rescues CFTR protein function and has made a significant impact on the lives of many people with CF. In Europe, ETI is currently available for people with CF who have at least one F508del...

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PMID: 39523185
DOI: 10.1016/j.jcf.2024.09.019

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CFTR

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Elexacaftor/tezacaftor/ivacaftor efficacy in intestinal organoids with rare CFTR variants in comparison to CFTR-F508del and CFTR-wild type controls.PMID 39523185Read ↗

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