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

Abstract

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 <i>in vitro</i> model to bring treatment to patients carrying rare <i>CFTR</i> mutations.