Ontology highlight
ABSTRACT:
SUBMITTER: Bose SJ
PROVIDER: S-EPMC7052731 | biostudies-literature | 2020 Mar
REPOSITORIES: biostudies-literature
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society 20200103
The treatment of cystic fibrosis (CF) has been transformed by orally-bioavailable small molecule modulators of the cystic fibrosis transmembrane conductance regulator (CFTR), which restore function to CF mutants. However, CFTR modulators are not available to all people with CF and better modulators are required to prevent disease progression. Here, we review selectively recent advances in CFTR folding, function and pharmacology. We highlight ensemble and single-molecule studies of CFTR folding, ...[more]