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 …

Standard follow-up and symptomatic treatment have allowed most patients with cystic
fibrosis to live to young adulthood. However, many patients still die prematurely from …

Small molecule chaperones have been exploited as therapeutics for the hundreds of
diseases caused by protein misfolding. The most successful examples are the CFTR …

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 …

An improved understanding of the cystic fibrosis (CF) transmembrane conductance regulator
(CFTR) protein structure and the consequences of CFTR gene mutations have allowed the …

With the discovery of the CFTR gene in 1989, the search for therapies to improve the basic
defects of cystic fibrosis (CF) commenced. Pharmacological manipulation provides the …

Available corrector drugs are unable to effectively rescue the folding defects of CFTR-ΔF508
(or CFTR-F508del), the most common disease-causing mutation of the cystic fibrosis …

Protein misfolding is involved in a large number of diseases, among which cystic fibrosis.
Complex intra-and inter-domain folding defects associated with mutations in the cystic …

Mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene cause a
characteristic defect in epithelial ion transport that plays a central role in the pathogenesis of …

CFTR biogenesis starts with its co-translational insertion into the membrane of endoplasmic
reticulum and folding of the cytosolic domains, towards the acquisition of a fully folded …