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

Conclusions 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 in vitro model to bring treatment to patients carrying rare CFTR mutations.

http://erj.ersjournals.com/cgi/content/short/57/1/1902426?rss=1

Source: European Respiratory Journal - January 5, 2021 Category: Respiratory Medicine Authors: Ramalho, A. S., Fürstova, E., Vonk, A. M., Ferrante, M., Verfaillie, C., Dupont, L., Boon, M., Proesmans, M., Beekman, J. M., Sarouk, I., Vazquez Cordero, C., Vermeulen, F., De Boeck, K., on behalf of the Belgian Organoid Project Tags: Lung biology and experimental studies, CF and non-CF bronchiectasis Original Articles: Cystic fibrosis and basic science Source Type: research