Ontology highlight
ABSTRACT:
SUBMITTER: Csanady L
PROVIDER: S-EPMC6594753 | biostudies-literature | 2019 Jun
REPOSITORIES: biostudies-literature
Csanády László L Töröcsik Beáta B
eLife 20190617
The devastating inherited disease cystic fibrosis (CF) is caused by mutations of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) anion channel. The recent approval of the CFTR potentiator drug ivacaftor (Vx-770) for the treatment of CF patients has marked the advent of causative CF therapy. Currently, thousands of patients are being treated with the drug, and its molecular mechanism of action is under intensive investigation. Here we determine the solubility profile and true stimu ...[more]