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- W3192016578 abstract "Superior efficacy of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) over tezacaftor/ivacaftor (TEZ/IVA) in people with cystic fibrosis (CF) and Phe508del/Phe508del genotype was shown in clinical trials. We utilized intestinal organoid approach to compare in vitro responses to these 2 CFTR modulator drug combinations and to check potential inter-individual variability in therapeutic response to the triple combination. Organoids from 17 subjects with Phe508del/Phe508del were screened with forskolin induced swelling assay. Significantly larger swelling, when exposed to ELX/TEZ/IVA as compared to TEZ/IVA, was observed in 16 of them. However, 1 sample showed no additional effect of ELX. The finding of unique CFTR variants in this sample indicates that genetic traits other than CF-causing CFTR mutation are worth exploring as they may have an impact on the definitive modulator drug response." @default.
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- W3192016578 date "2022-03-01" @default.
- W3192016578 modified "2023-10-18" @default.
- W3192016578 title "Response to elexacaftor/tezacaftor/ivacaftor in intestinal organoids derived from people with cystic fibrosis" @default.
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- W3192016578 doi "https://doi.org/10.1016/j.jcf.2021.07.006" @default.
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