Ageing is a major risk factor for the development of many diseases, prominently including
neurodegenerative disorders such as Alzheimer disease and Parkinson disease. A hallmark …

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 …

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

Little is known about the relative importance of monocyte and tissue-resident macrophages
in the development of lung fibrosis. We show that specific genetic deletion of monocyte …

Background Lumacaftor and ivacaftor combination treatment showed efficacy in patients
aged 12 years or older with cystic fibrosis homozygous for F508del-cystic fibrosis …

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 …

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 …

The selective degradation of many short-lived proteins in eukaryotic cells is carried out by
the ubiquitin system. In this pathway, proteins are targeted for degradation by covalent …

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

Intracellular deposition of misfolded protein aggregates into ubiquitin-rich cytoplasmic
inclusions is linked to the pathogenesis of many diseases. Why these aggregates form …