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CFTR Function Restoration upon Elexacaftor/Tezacaftor/Ivacaftor Treatment in Patient-Derived Intestinal Organoids with Rare CFTR Genotypes
Cystic fibrosis (CF) is caused by mutations in the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) gene. The combination of the CFTR modulators elexacaftor, tezacaftor, and ivacaftor (ETI) enables the effective rescue of CFTR function in people with the most prevalent F508del mutation. Ho...
Autores principales: | Lefferts, Juliet W., Bierlaagh, Marlou C., Kroes, Suzanne, Nieuwenhuijze, Natascha D. A., Sonneveld van Kooten, Heleen N., Niemöller, Paul J., Verburg, Tibo F., Janssens, Hettie M., Muilwijk, Danya, van Beuningen, Sam F. B., van der Ent, Cornelis K., Beekman, Jeffrey M. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2023
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10572896/ https://www.ncbi.nlm.nih.gov/pubmed/37833986 http://dx.doi.org/10.3390/ijms241914539 |
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