Executive summary The past six decades have seen remarkable improvements in health
outcomes for people with cystic fibrosis, which was once a fatal disease of infants and young …

Cystic fibrosis (CF) is a lethal inherited disease caused by mutations in the CF
transmembrane conductance regulator (CFTR) gene, which result in impairment of CFTR …

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

We recently established conditions allowing for long-term expansion of epithelial organoids
from intestine, recapitulating essential features of the in vivo tissue architecture. Here we …

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 …

Control of protein levels by nucleic-acid-based technologies has proven to be a useful
research tool but lacks the advantages of small molecules with respect to cell permeability …

Cystic fibrosis (CF) is caused by mutations in the CF transmembrane conductance regulator
(CFTR) gene that impair the function of CFTR, an epithelial chloride channel required for …

Cystic fibrosis (CF) remains the most common life-shortening hereditary disease in white
populations, with high morbidity and mortality related to chronic airway mucus obstruction …

Cystic fibrosis (CF) is a fatal genetic disease caused by mutations in the gene encoding the
CF transmembrane conductance regulator (CFTR), a protein kinase A (PKA)-activated …

The folding/misfolding and pharmacological rescue of multidomain ATP-binding cassette
(ABC) C-subfamily transporters, essential for organismal health, remain incompletely …