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Updated: Dec 28, 2025
The Ex Vivo Culture and Pattern Recognition Receptor Stimulation of Mouse Intestinal Organoids
Published on: May 18, 2016
R117H-CFTR function and response to VX-770 correlate with mRNA and protein expression in intestinal organoids
Peter Van Mourik1, Paul van Haaren2, Evelien Kruisselbrink2
1Department of Pediatric Pulmonology, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, Utrecht, the Netherlands, member of ERN-LUNG.
Introduction:
Variability in disease severity and CFTR modulator responses exists between patients with identical CFTR genotypes. Here, we characterized transcription, translation and function of R117H-CFTR using intestinal organoids and correlated them with in vitro responses to ivacaftor (VX-770).
Methods:
Organoids were generated from individuals possessing at least one R117H-CFTR allele. The forskolin-induced swelling (FIS) assay was used to measure CFTR function and response to VX-770 treatment. R117H-CFTR protein and mRNA expression levels were determined in parallel and Pearson's correlation coefficients were assessed.
Results:
Variability in R117H-CFTR FIS responses was observed and correlated significantly with mRNA and protein expression. Response to VX-770 treatment in organoids correlated with mRNA and protein expression as well.
Conclusions:
Our results indicate that gene expression, protein expression and CFTR function are strongly correlated in organoids from people with CFTR-R117H-7T/9T, which may suggest that CFTR gene expression may have consequences for CF diagnosis, prognosis and therapeutic benefit.
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