Response to elexacaftor/tezacaftor/ivacaftor in intestinal organoids derived from people with cystic fibrosis
The result's identifiers
Result code in IS VaVaI
<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00216208%3A11130%2F22%3A10429908" target="_blank" >RIV/00216208:11130/22:10429908 - isvavai.cz</a>
Alternative codes found
RIV/00064203:_____/22:10429908
Result on the web
<a href="https://verso.is.cuni.cz/pub/verso.fpl?fname=obd_publikace_handle&handle=Q.mKzmQy7P" target="_blank" >https://verso.is.cuni.cz/pub/verso.fpl?fname=obd_publikace_handle&handle=Q.mKzmQy7P</a>
DOI - Digital Object Identifier
<a href="http://dx.doi.org/10.1016/j.jcf.2021.07.006" target="_blank" >10.1016/j.jcf.2021.07.006</a>
Alternative languages
Result language
angličtina
Original language name
Response to elexacaftor/tezacaftor/ivacaftor in intestinal organoids derived from people with cystic fibrosis
Original language description
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.
Czech name
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Czech description
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Classification
Type
J<sub>imp</sub> - Article in a specialist periodical, which is included in the Web of Science database
CEP classification
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OECD FORD branch
30209 - Paediatrics
Result continuities
Project
<a href="/en/project/NU20-07-00049" target="_blank" >NU20-07-00049: Intestinal organoids as an in-vitro model for predicting the response to therapies correcting molecular defects in cystic fibrosis</a><br>
Continuities
P - Projekt vyzkumu a vyvoje financovany z verejnych zdroju (s odkazem do CEP)
Others
Publication year
2022
Confidentiality
S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů
Data specific for result type
Name of the periodical
Journal of Cystic Fibrosis
ISSN
1569-1993
e-ISSN
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Volume of the periodical
21
Issue of the periodical within the volume
2
Country of publishing house
NL - THE KINGDOM OF THE NETHERLANDS
Number of pages
3
Pages from-to
243-245
UT code for WoS article
000820193000025
EID of the result in the Scopus database
2-s2.0-85111676267