Article
Forskolin induced swelling (FIS) assay in intestinal organoids to guide eligibility for compassionate use treatment in a CF patient with a rare genotype.
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society - 1 Mar 2022
Aalbers B L, Brunsveld J E, van der Ent C K, van den Eijnden J C, Beekman J M, Heijerman H G M
Abstract excerpt
BACKGROUND: Forskolin-induced swelling of patient-derived organoids has been used to measure patient-specific CFTR function and CFTR modulator response. We present a case where CFTR function assessment in intestinal organoids was decisive for a patients' acceptance to a compassionate use program. CASE DESCRIPTION: A 56 years old female with cystic fibrosis compound heterozygous for F508del and a rare CFTR allele...
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