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 …

Despite lipid nanoparticles'(LNPs) success in the effective and safe delivery of mRNA
vaccines, an inhalation-based mRNA therapy for lung diseases remains challenging. LNPs …

Background Elexacaftor–tezacaftor–ivacaftor is a small-molecule cystic fibrosis
transmembrane conductance regulator (CFTR) modulator regimen shown to be efficacious …

The autosomal recessive disease cystic fibrosis (CF) was once untreatable and deadly in
childhood, but now most patients survive to adulthood. Many countries have instituted CF …

Background The next-generation cystic fibrosis transmembrane conductance regulator
(CFTR) corrector VX-659, in triple combination with tezacaftor and ivacaftor (VX-659 …

Cystic fibrosis (CF) is a recessive genetic disease caused by mutations in a gene encoding
a protein called Cystic Fibrosis Transmembrane Conductance Regulator (CFTR). The CFTR …

Cystic fibrosis (CF) is a potentially fatal genetic disease that causes serious lung damage.
With time, researchers have a more complete understanding of the molecular-biological …

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 …

The cystic fibrosis transmembrane conductance regulator (CFTR) belongs to the ATP
binding cassette (ABC) transporter superfamily but functions as an anion channel crucial for …