The accumulation of misfolded proteins in the endoplasmic reticulum (ER) leads to ER
stress, resulting in activation of the unfolded protein response (UPR) that aims to restore …

Importance Cystic fibrosis, a genetic disorder defined by variants in the cystic fibrosis
transmembrane conductance regulator (CFTR) gene, affects more than 30 000 individuals in …

Background Time has seen management for Cystic Fibrosis (CF) advance drastically, most
recently in the development of the disease-modifying triple combination therapy …

The predominant mutation causing cystic fibrosis, a deletion of phenylalanine 508 (Δ508) in
the cystic fibrosis transmembrane conductance regulator (CFTR), leads to severe defects in …

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

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

Background Around 20% of people with cystic fibrosis (pwCF) do not have access to the
triple combination elexacaftor/tezacaftor/ivacaftor (ETI) in Europe because they do not carry …

Following discovery of the cystic fibrosis transmembrane conductance regulator (CFTR)
gene in 1989 and subsequent elucidation of the varied CFTR protein abnormalities that …

Cystic fibrosis (CF) is caused by mutations in the cystic fibrosis transmembrane conductance
regulator (CFTR), which lead to early death due to progressive lung disease. The …

Pyrazole is a five-membered heterocycle bearing two adjacent nitrogen atoms. Both
pharmaceutical agents and natural products with pyrazole as a nucleus have exhibited a …