Results 41 to 50 of about 10,277 (199)

Lived experiences of individuals with cystic fibrosis on CFTR-modulators

BMC Pulmonary Medicine, 2022
Background CFTR-modulators are a category of drugs that facilitate trafficking and opening of the abnormal CFTR protein in individuals with cystic fibrosis (CF) who have certain genetic mutations.
Annelise Page, Aaron Goldenberg, Anne L. Matthews   +2 more
doaj   +1 more source

Chronic inflammation in CF airways - a persistent issue for A20 [PDF]

, 2016
Cystic Fibrosis (CF) is characterised by prolonged and exaggerated airways inflammation. Despite recent developments to overcome the underlying functional defect in CFTR (cystic fibrosis transmembrane conductance regulator), there is still an unmet need ...
El Banna, Amal, Ennis, Madeleine, McCollum, Kirsten, Schock, Bettina   +3 more
core   +1 more source

Novel CFTR modulator combinations maximise rescue of G85E and N1303K in rectal organoids

ERJ Open Research, 2022
Introduction Cystic fibrosis (CF) is a severe monogenic disorder caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
Marjolein M. Ensinck, Liesbeth De Keersmaecker, Anabela S. Ramalho, Senne Cuyx, Stephanie Van Biervliet, Lieven Dupont, Frauke Christ, Zeger Debyser, François Vermeulen, Marianne S. Carlon   +9 more
doaj   +1 more source

From CFTR biology toward combinatorial pharmacotherapy:expanded classification of cystic fibrosis mutations [PDF]

, 2016
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 phenotypes.
Avramescu, Radu G., Balch, William E., Brodsky, Jeffrey L., Cai, Zhiwei, Chiang, Annette N., Cutting, Garry R., Cyr, Douglas M., Frizzell, Raymond A., Guggino, William B., Hong, Jeong S., Houck, Scott A., Lukacs, Gergely L., Peters, Kathryn W., Pollard, Harvey B., Sheppard, David, Skach, William R., Sorscher, Eric J., Veit, Gudio   +17 more
core   +3 more sources

Bioengineered 3D hPSC‐Cholangiocyte Ducts With Physiological Signals for Biliary Disease Modeling

Advanced Healthcare Materials, EarlyView.
Tian and colleagues generated a bioengineered bile duct from human pluripotent stem cell (hPSC)‐derived intrahepatic cholangiocytes within a high‐throughput, 384‐well platform to systematically examine the influence of biliary physiological signals including fluid flow, stromal cells and bile acids, and models intrahepatic biliary disease progression ...
Britney Tian, Mina Ogawa, Manami Kondo, Gabrielle Langeveld, Ling Jun Huan, Feng Zhang, Andrew Hollinger, Sara Deir, Christine Bear, Boyang Zhang, Shinichiro Ogawa   +10 more
wiley   +1 more source

Impact of CFTR modulators on exercise capacity in adolescents with cystic fibrosis

ERJ Open Research
Background Exercise capacity is an independent predictor of clinical worsening in cystic fibrosis (CF). There is limited evidence of the impact of cystic fibrosis transmembrane conductance regulator (CFTR) modulators on exercise capacity in children with
Molla Imaduddin Ahmed, Naomi Dayman, Natalie Blyth, Joe Madge, Erol Gaillard   +4 more
doaj   +1 more source

Plasma membrane-specific interactome analysis reveals calpain 1 as a druggable modulator of rescued Phe508del-CFTR cell surface stability [PDF]

, 2019
Cystic fibrosis (CF) is a genetic disease caused by mutations in the gene encoding CF transmembrane conductance regulator (CFTR), a chloride channel normally expressed at the surface of epithelial cells.
Amaral, Margarida D., Barros, Patrícia, Matos, Ana Margarida, Matos, Paulo, Pepperkok, Rainer, Pinto, Francisco R.   +5 more
core  

Attenuation of Phosphorylation-dependent activation of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) by disease-causing mutations at the transmission interface [PDF]

, 2016
Cystic fibrosis transmembrane conductance regulator (CFTR) is a multidomain membrane protein that functions as a phosphorylation-regulated anion channel. The interface between its two cytosolic nucleotide binding domains and coupling helices conferred by
Bear, C.E., Chin, S., Eckford, P.D.W., Miles, Andrew J., Molinski, S., Wallace, Bonnie A., Yang, D.   +6 more
core   +1 more source

Respiratory Organ‐on‐a‐Chip for Disease Modeling: From Architecture to Functional Integration

Advanced Healthcare Materials, EarlyView.
Respiratory organ‐on‐a‐chip (ROC) models capture key mechanical and cellular cues of the human respiratory system, enabling quantitative dissection of disease mechanisms. This review links ROC architectures to disease modeling, functional integration, and commercialization, and proposes a decision framework that aligns model complexity with mechanistic
Jinzhuo Hu, Yongjie Tang, Sidi Liu, Tao Xu, Qian Liu   +4 more
wiley   +1 more source

Allergic Bronchopulmonary Aspergillosis (ABPA) in the Era of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Modulators

Journal of Fungi
Allergic bronchopulmonary aspergillosis (ABPA) is a hypersensitivity disease caused by Aspergillus fumigatus (Af), prevalent in persons with cystic fibrosis (CF) or asthma.
Paulami Chatterjee, Carson Tyler Moss, Sarah Omar, Ekroop Dhillon, Carlos Daniel Hernandez Borges, Alan C. Tang, David A. Stevens, Joe L. Hsu   +7 more
doaj   +1 more source