The substantial investments in human genetics and genomics made over the past three
decades were anticipated to result in many innovative therapies. Here we investigate the …

More than 2000 mutations in the cystic fibrosis transmembrane conductance regulator
(CFTR) have been described that confer a range of molecular cell biological and functional …

Extended-connectivity fingerprints (ECFPs) are a novel class of topological fingerprints for
molecular characterization. Historically, topological fingerprints were developed for …

Advances over the past 25 years have revealed much about how the structural properties of
membranes and associated proteins are linked to the thermodynamics and kinetics of …

This report describes a number of substructural features which can help to identify
compounds that appear as frequent hitters (promiscuous compounds) in many biochemical …

Cystic fibrosis (CF) is caused by mutations in the CF transmembrane regulator (CFTR) that
result in reduced anion conductance at the apical membrane of secretory epithelia …

Cystic fibrosis (CF), the most common lethal genetic disease in the Caucasian population, is
caused by loss-of-function mutations of the CF transmembrane conductance regulator …

Cystic fibrosis (CF) is caused by mutations in the CF transmembrane conductance regulator
(CFTR). Newly developed “correctors” such as lumacaftor (VX-809) that improve CFTR …

Mutations in the gene coding for the cystic fibrosis transmembrane conductance regulator
(CFTR) epithelial anion channel cause cystic fibrosis (CF). The multidomain integral …

Cystic fibrosis (CF) is a fatal genetic disease caused by mutations in cftr, a gene encoding a
PKA-regulated Cl− channel. The most common mutation results in a deletion of …