Response to elexacaftor/tezacaftor/ivacaftor in intestinal organoids derived from people with cystic fibrosis.
J Cyst Fibros
; 21(2): 243-245, 2022 03.
Article
en En
| MEDLINE
| ID: mdl-34348870
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.
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Base de datos:
MEDLINE
Asunto principal:
Organoides
/
Fibrosis Quística
Idioma:
En
Revista:
J Cyst Fibros
Año:
2022
Tipo del documento:
Article