Forskolin-induced swelling in intestinal organoids: an in vitro assay for assessing drug response in cystic fibrosis patients
SF Boj, AM Vonk, M Statia, J Su, JF Dekkers… - JoVE (Journal of …, 2017 - jove.com
Recently-developed cystic fibrosis transmembrane conductance regulator (CFTR)-
modulating drugs correct surface expression and/or function of the mutant CFTR channel in
subjects with cystic fibrosis (CF). Identification of subjects that may benefit from these drugs
is challenging because of the extensive heterogeneity of CFTR mutations, as well as other
unknown factors that contribute to individual drug efficacy. Here, we describe a simple and
relatively rapid assay for measuring individual CFTR function and response to CFTR …
modulating drugs correct surface expression and/or function of the mutant CFTR channel in
subjects with cystic fibrosis (CF). Identification of subjects that may benefit from these drugs
is challenging because of the extensive heterogeneity of CFTR mutations, as well as other
unknown factors that contribute to individual drug efficacy. Here, we describe a simple and
relatively rapid assay for measuring individual CFTR function and response to CFTR …
