Impact of Airway Inflammation on the Efficacy of CFTR Modulators [PDF]
Cells, 2021 Defective CFTR biogenesis and activity in cystic fibrosis airways leads to airway dehydration and impaired mucociliary clearance, resulting in chronic airway infection and inflammation.
Carla M. P. Ribeiro, Martina Gentzsch
doaj +3 more sources
Mitochondrial Fragmentation Induced by the CFTR Modulators Lumacaftor and Ivacaftor in Immortalized Cystic Fibrosis Cell Lines [PDF]
CellsCystic fibrosis (CF) is an autosomal recessive disease caused by mutations in the CFTR gene, which encodes a cAMP-activated chloride channel essential for epithelial function.
Camila Dib +8 more
doaj +2 more sources
Medication adherence to CFTR modulators in patients with cystic fibrosis: a systematic review [PDF]
European Respiratory ReviewBackground In the last decade, a fundamental shift in the treatment of cystic fibrosis (CF) took place due to the introduction of CF transmembrane conductance regulator (CFTR) modulators.
Carina M.E. Hansen +5 more
doaj +2 more sources
Epithelial responses to CFTR modulators are improved by inflammatory cytokines and impaired by antiinflammatory drugs [PDF]
JCI InsightCystic fibrosis (CF) is a genetic disorder that disrupts CF transmembrane conductance regulator (CFTR) anion channels and impairs airway host defenses. Airway inflammation is ubiquitous in CF, and suppressing it has generally been considered to improve ...
Tayyab Rehman +4 more
doaj +2 more sources
Effect of CFTR modulators on glucose homeostasis in children and young adults with cystic fibrosis-related diabetes: a systematic review [PDF]
Frontiers in EndocrinologyIntroductionCystic fibrosis (CF) is an autosomal recessive disorder caused by mutations in the CFTR gene, leading to impaired chloride transport, thickened mucus, and multiorgan dysfunction. Among its complications, cystic fibrosis-related diabetes (CFRD)
Paola Giordano +6 more
doaj +2 more sources
CFTR modulators response of S737F and T465N CFTR variants on patient-derived rectal organoids [PDF]
Orphanet Journal of Rare DiseasesBackground Predictions based on patient-derived materials of CFTR modulators efficacy have been performed lately in patient-derived cells, extending FDA-approved drugs for CF patients harboring rare variants.
Karina Kleinfelder +6 more
doaj +2 more sources
Pharmacological Responses of the G542X-CFTR to CFTR Modulators
Frontiers in Molecular Biosciences, 2022 Cystic fibrosis (CF) is a lethal hereditary disease caused by loss-of-function mutations of the chloride channel cystic fibrosis transmembrane conductance regulator (CFTR). With the development of small-molecule CFTR modulators, including correctors that facilitate protein folding and expression and potentiators that promote channel activity, about 90%
Xinxiu Fang +3 more
openaire +3 more sources
Frontiers in Molecular Biosciences, 2023 Most of the 2,100 CFTR gene variants reported to date are still unknown in terms of their disease liability in Cystic Fibrosis (CF) and their molecular and cellular mechanism that leads to CFTR dysfunction.
Violeta Railean +7 more
doaj +1 more source
Cells, 2023 People with cystic fibrosis (pwCF) suffer from chronic and recurring bacterial lung infections that begin very early in life and contribute to progressive lung failure.
Deborah M. Cholon +11 more
doaj +1 more source
Mutual Effects of Single and Combined CFTR Modulators and Bacterial Infection in Cystic Fibrosis
Microbiology Spectrum, 2023 Cystic fibrosis transmembrane conductance regulator (CFTR) modulators improve clinical outcomes with varied efficacies in patients with CF. However, the mutual effects of CFTR modulators and bacterial adaptation, together with antibiotic regimens, can ...
Cristina Cigana +9 more
doaj +1 more source