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 …

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 children. However, …

… Correspondence and requests for reprints should be addressed to Jane C. Davies, MD,
MBCh.B., Department of Gene Therapy, Imperial College London, Emmanuel Kaye Building, …

Background Lung delivery of plasmid DNA encoding the CFTR gene complexed with a
cationic liposome is a potential treatment option for patients with cystic fibrosis. We aimed to …

Background Cystic fibrosis is an autosomal recessive disease caused by mutations in the
CFTR gene that lead to progressive respiratory decline. Some mutant CFTR proteins show …

Pseudomonas aeruginosa is the major pathogen in the cystic fibrosis (CF) lung. Prevalence
is high and, once acquired, chronic infection will almost always ensue. Several hypotheses …

Pulmonary delivery of plasmid DNA (pDNA)/cationic liposome complexes is associated with
an acute unmethylated CG dinucleotide (CpG)-mediated inflammatory response and brief …

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

Background Ivacaftor has been shown to be a safe, effective treatment for cystic fibrosis in
patients aged 6 years or older with a CFTR gating mutation. We aimed to assess the safety, …

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