Correction of CFTR function in intestinal organoids to guide treatment of cystic fibrosis.

Given the vast number of cystic fibrosis transmembrane conductance regulator ( To study CFTR function in organoids of subjects with common and rare Intestinal organoids were grown from rectal biopsies in a cohort of 97 subjects with CF. Residual CFTR function was measured by quantifying organoid swelling induced by forskolin and response to modulators by quantifying organoid swelling induced by CFTR correctors, potentiator and their combination. Organoid data were correlated with clinical data from the literature. Across 28 genotypes, residual CFTR function correlated (r Measurements of residual CFTR function and rescue of function by CFTR modulators in intestinal organoids correlate closely with clinical data. Our results for reference genotypes concur with previous results. CFTR function measured in organoids can be used to guide precision medicine in patients with CF, positioning organoids as a potential

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Conflict of interest: A.S. Ramalho has nothing to disclose. Conflict of interest: E. Fürstová has nothing to disclose. Conflict of interest: A.M. Vonk has nothing to disclose. Conflict of interest: M. Ferrante reports grants and personal fees from Amgen, grants, personal fees and non-financial support from Biogen, Janssen, Pfizer and Takeda, personal fees and non-financial support from Boehringer Ingelheim, MSD, Falk and Ferring, personal fees from Sandoz, Lamepro, and Mylan, outside the submitted work. Conflict of interest: C. Verfaillie has nothing to disclose. Conflict of interest: L. Dupont has nothing to disclose. Conflict of interest: M. Boon is a member of the European Reference Network for Rare Respiratory Diseases (ERN-LUNG) – Project ID number 739546. Conflict of interest: M. Proesmans has nothing to disclose. Conflict of interest: J.M. Beekman reports personal fees from various industries (Vertex, Proteostasis, Teva, others) for costs related to conference presentations, outside the submitted work; and has a patent WO2013093812A3 with royalties paid by Hubrecht Organoid Technology. Conflict of interest: I. Sarouk has nothing to disclose. Conflict of interest: C. Vazquez Cordero has nothing to disclose. Conflict of interest: F. Vermeulen has nothing to disclose. Conflict of interest: K. De Boeck has provided consultancy for Boehringer, Protalix, Raptor, Novabiotics, Eloxx and Chiesi, has been member of steering committees/advisory boards and been PI in studies for Vertex, has provided consultancy and been PI in studies for Galapagos, and has received speaker fees from Teva, outside the submitted work.