CFTR Function Restoration upon Elexacaftor/Tezacaftor/Ivacaftor Treatment in Patient-Derived Intestinal Organoids with Rare <i>CFTR</i> Genotypes

CFTR Function Restoration upon Elexacaftor/Tezacaftor/Ivacaftor Treatment in Patient-Derived Intestinal Organoids with Rare <i>CFTR</i> Genotypes

Cystic fibrosis (CF) is caused by mutations in the <i>Cystic Fibrosis Transmembrane conductance Regulator</i> (<i>CFTR</i>) gene. The combination of the CFTR modulators elexacaftor, tezacaftor, and ivacaftor (ETI) enables the effective rescue of CFTR function in people with t...

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Bibliographic Details
Main Authors: Juliet W. Lefferts, Marlou C. Bierlaagh, Suzanne Kroes, Natascha D. A. Nieuwenhuijze, Heleen N. Sonneveld van Kooten, Paul J. Niemöller, Tibo F. Verburg, Hettie M. Janssens, Danya Muilwijk, Sam F. B. van Beuningen, Cornelis K. van der Ent, Jeffrey M. Beekman
Format: Article
Language:English
Published: MDPI AG 2023-09-01
Series:International Journal of Molecular Sciences
Subjects:
cystic fibrosis
CFTR modulator therapy
intestinal organoids
elexacaftor/tezacaftor/ivacaftor
theratyping
theranostics
Online Access:https://www.mdpi.com/1422-0067/24/19/14539

Internet

https://www.mdpi.com/1422-0067/24/19/14539

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