Article
FDA-approved drug screening in patient-derived organoids demonstrates potential of drug repurposing for rare cystic fibrosis genotypes.
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society - 1 May 2023
de Poel E, Spelier S, Hagemeijer M C, van Mourik P, Suen S W F, Vonk A M, Brunsveld J E, Ithakisiou G N, Kruisselbrink E, Oppelaar H, Berkers G, de Winter de Groot K M, Heida-Michel S, Jans S R, van Panhuis H, Bakker M, van der Meer R, Roukema J, Dompeling E, Weersink E J M, Koppelman G H, Blaazer A R, Muijlwijk-Koezen J E, van der Ent C K, Beekman J M
Abstract excerpt
BACKGROUND: Preclinical cell-based assays that recapitulate human disease play an important role in drug repurposing. We previously developed a functional forskolin induced swelling (FIS) assay using patient-derived intestinal organoids (PDIOs), allowing functional characterization of CFTR, the gene mutated in people with cystic fibrosis (pwCF). CFTR function-increasing pharmacotherapies have revolutionized...
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