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 …

Signal transduction cascades efficiently transmit chemical and/or physical signals from the
extracellular environment to intracellular compartments, thereby eliciting an appropriate …

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 …

Background Cystic fibrosis transmembrane conductance regulator (CFTR) modulators
correct the basic defect caused by CFTR mutations. Improvements in health outcomes have …

Background Cystic fibrosis is caused by mutations in the gene encoding the cystic fibrosis
transmembrane conductance regulator (CFTR) protein, and nearly 90% of patients have at …

Prime editing (PE) enables precise and versatile genome editing without requiring double-
stranded DNA breaks. Here we describe the systematic optimization of PE systems to …

Background Combination treatment with the cystic fibrosis transmembrane conductance
regulator (CFTR) modulators tezacaftor (VX-661) and ivacaftor (VX-770) was designed to …

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 Cystic fibrosis is a life-limiting disease that is caused by defective or deficient
cystic fibrosis transmembrane conductance regulator (CFTR) protein activity. Phe508del is …

The cystic fibrosis transmembrane conductance regulator (CFTR) is an anion channel
evolved from the ATP-binding cassette (ABC) transporter family. In this study, we determined …