Pharmacological Modulation of Ion Channels for the Treatment of Cystic Fibrosis.

ABSTRACT: Cystic fibrosis (CF) is a life-shortening monogenic disease caused by mutations in the gene encoding the CF transmembrane conductance regulator (CFTR) protein, an anion channel that transports chloride and bicarbonate across epithelia. Despite clinical progress in delaying disease progression with symptomatic therapies, these individuals still develop various chronic complications in lungs and other organs, which significantly restricts their life expectancy and quality of life. The development of high-throughput assays to screen drug-like compound libraries have enabled the discovery of highly effective CFTR modulator therapies. These novel therapies target the primary defect underlying CF and are now approved for clinical use for individuals with specific CF genotypes. However, the clini

SUBMITTER: Pinto MC 

PROVIDER: S-EPMC8316759 | biostudies-literature | 2021

REPOSITORIES: biostudies-literature