Different CFTR modulator combinations downregulate inflammation differently in cystic fibrosis
eLife, 2020 Previously, we showed that serum and monocytes from patients with CF exhibit an enhanced NLRP3-inflammasome signature with increased IL-18, IL-1β, caspase-1 activity and ASC speck release (Scambler et al. eLife 2019).
H. Jarosz-Griffiths +13 more
semanticscholar +1 more source
Progress in precision medicine in cystic fibrosis: a focus on CFTR modulator therapy
Breathe, 2021 The genetic multisystem condition cystic fibrosis (CF) has seen a paradigm shift in therapeutic approaches within the past decade. Since the first clinical descriptions in the 1930s, treatment advances had focused on the downstream consequences of a ...
D. Tewkesbury, R. Robey, P. Barry
semanticscholar +1 more source
Cytokine-Regulation of Na+-K+-Cl− Cotransporter 1 and Cystic Fibrosis Transmembrane Conductance Regulator—Potential Role in Pulmonary Inflammation and Edema Formation [PDF]
, 2017 Pulmonary edema, a major complication of lung injury and inflammation, is defined as accumulation of extravascular fluid in the lungs leading to impaired diffusion of respiratory gases.
Sarah Weidenfeld, Wolfgang M. Kuebler
core +1 more source
Impact of CFTR modulator use on outcomes in people with severe cystic fibrosis lung disease
European Respiratory Review, 2020 Drug compounds that augment the production and activity of the cystic fibrosis (CF) transmembrane regulator (CFTR) have revolutionised CF care. Many adults and some children with CF suffer advanced and severe lung disease or await lung transplantation ...
M. Shteinberg, J. Taylor-Cousar
semanticscholar +1 more source
Fertility, Pregnancy and Lactation Considerations for Women with CF in the CFTR Modulator Era
Journal of Personalized Medicine, 2021 Cystic fibrosis (CF) is an autosomal recessive genetic disorder impacting approximately 80,000 people of all races and ethnicities world-wide. CF is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene which encodes ...
R. Jain, J. Taylor-Cousar
semanticscholar +1 more source
Rapid therapeutic advances in CFTR modulator science [PDF]
Pediatric Pulmonology, 2018 AbstractCystic fibrosis (CF) is an autosomal recessive genetic disease caused by variants in the gene encoding the cystic fibrosis transmembrane conduction regulator (CFTR) protein. Loss of CFTR function disrupts chloride, bicarbonate and regulation of sodium transport, producing a cascade of mucus obstruction, inflammation, pulmonary infection, and ...
openaire +2 more sources
Strategies to Improve the Lipophilicity of Hydrophilic Macromolecular Drugs
Advanced Healthcare Materials, EarlyView.Hydrophilic macromolecular drugs can be successfully lipidized by covalent attachment of lipids, by hydrophobic ion pairing with negatively or positively charged surfactants, and by dry or wet reverse micelle formation. Lipophilicity enhancement of hydrophilic macromolecules has several benefits including stability and bioavailability improvement ...
Sera Lindner +8 more
wiley +1 more source
Frontiers in Molecular Biosciences, 2023 Background: Cystic fibrosis (CF) is caused by a wide spectrum of mutations in the CF transmembrane conductance regulator (CFTR) gene, with some leading to non-classical clinical presentations.
Katelin M. Allan +29 more
doaj +1 more source
Bioactive Thymosin Alpha-1 Does Not Influence F508del-CFTR Maturation and Activity. [PDF]
, 2019 Deletion of phenylalanine 508 (F508del) in the cystic fibrosis transmembrane conductance regulator (CFTR) anion channel is the most frequent mutation causing cystic fibrosis (CF). F508del-CFTR is misfolded and prematurely degraded. Recently thymosin a-1 (
Armirotti, Andrea +13 more
core
Some gating potentiators, including VX-770, diminish ΔF508-CFTR functional expression. [PDF]
, 2014 Cystic fibrosis (CF) is caused by mutations in the CF transmembrane regulator (CFTR) that result in reduced anion conductance at the apical membrane of secretory epithelia.
Apaja, Pirjo M. +12 more
core +1 more source