Results 41 to 50 of about 1,459,532 (261)

Measurements of spontaneous CFTR-mediated ion transport without acute channel activation in airway epithelial cultures after modulator exposure

Scientific Reports, 2021
Quantitation of CFTR function in vitro is commonly performed by acutely stimulating then inhibiting ion transport through CFTR and measuring the resulting changes in transepithelial voltage (Vte) and current (ISC).
Heidi J. Nick, Pamela L. Zeitlin, Sangya Yadav, Preston E. Bratcher   +3 more
doaj   +1 more source

S945L-CFTR molecular dynamics, functional characterization and tezacaftor/ivacaftor efficacy in vivo and in vitro in matched pediatric patient-derived cell models

Frontiers in Pediatrics, 2022
Cystic Fibrosis (CF) results from over 400 different disease-causing mutations in the CF Transmembrane Conductance Regulator (CFTR) gene. These CFTR mutations lead to numerous defects in CFTR protein function.
Katelin M. Allan, Katelin M. Allan, Miro A. Astore, Laura K. Fawcett, Laura K. Fawcett, Laura K. Fawcett, Sharon L. Wong, Sharon L. Wong, Po-Chia Chen, Renate Griffith, Adam Jaffe, Adam Jaffe, Adam Jaffe, Serdar Kuyucak, Shafagh A. Waters, Shafagh A. Waters, Shafagh A. Waters, Shafagh A. Waters   +17 more
doaj   +1 more source

Increase of liver stiffness and altered bile acid metabolism after triple CFTR modulator initiation in children and young adults with cystic fibrosis

Liver international (Print), 2023
Novel cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies (elexacaftor/tezacaftor/ivacaftor—ETI) promise clinically significant and sustained improvements for patients with cystic fibrosis (CF).
A. Schnell, J. Jüngert, D. Klett, Hannah Hober, Natalie Kaiser, R. Ruppel, Annika Geppert, Christina Tremel, Julia Sobel, Erika Plattner, S. Schmitt-Grohé, S. Zirlik, D. Strobel, M. Neurath, F. Knieling, M. Rauh, J. Woelfle, André Hoerning, Adrian P. Regensburger   +18 more
semanticscholar   +1 more source

YES1 Kinase Mediates the Membrane Removal of Rescued F508del-CFTR in Airway Cells by Promoting MAPK Pathway Activation via SHC1

Biomolecules, 2023
Recent developments in CFTR modulator drugs have had a significant transformational effect on the treatment of individuals with Cystic Fibrosis (CF) who carry the most frequent F508del-CFTR mutation in at least one allele.
Patrícia Barros, Ana M. Matos, Paulo Matos, Peter Jordan   +3 more
doaj   +1 more source

Modulation of CFTR gating by permeant ions [PDF]

Journal of General Physiology, 2014
Cystic fibrosis transmembrane conductance regulator (CFTR) is unique among ion channels in that after its phosphorylation by protein kinase A (PKA), its ATP-dependent gating violates microscopic reversibility caused by the intimate involvement of ATP hydrolysis in controlling channel closure. Recent studies suggest a gating model featuring an energetic
Yeh, Han-I, Yeh, Jiunn-Tyng, Hwang, Tzyh-Chang   +2 more
openaire   +2 more sources

Validating organoid-derived human intestinal monolayers for personalized therapy in cystic fibrosis

Life Science Alliance, 2023
Ussing chamber studies of intestinal organoid monolayers compare favorably to established preclinical tools to assess individual responses to CFTR modulator drugs in people with cystic fibrosis.
Liron Birimberg-Schwartz, Wan Ip, Claire Bartlett, Julie Avolio, Annelotte M Vonk, Tarini Gunawardena, Kai Du, Mohsen Esmaeili, Jeffrey M Beekman, Johanna Rommens, Lisa Strug, Christine E Bear, Theo J Moraes, Tanja Gonska   +13 more
doaj   +1 more source

Weight increase in people with cystic fibrosis on CFTR modulator therapy is mainly due to increase in fat mass

Frontiers in Pharmacology, 2023
Background: Ivacaftor, the first CFTR modulator drug, leads to significant long-term improvement in lung function and weight gain. The mechanism as well as the long-term impact of ivacaftor on weight, resting energy expenditure (REE) and body composition
M. Mouzaki, Annie Dupuis, J. Avolio, K. Griffin, F. Ratjen, E. Tullis, T. Gonska   +6 more
semanticscholar   +1 more source

Mechanism-based corrector combination restores ΔF508-CFTR folding and function. [PDF]

, 2013
The most common cystic fibrosis mutation, Delta F508 in nucleotide binding domain 1 (NBD1), impairs cystic fibrosis transmembrane conductance regulator (CFTR)-coupled domain folding, plasma membrane expression, function and stability. VX-809, a promising
Okiyoneda, T., Beekman, Jeffrey, Verkman, Alan, Soya, N., Bagdany, Miklos, Dekkers, J. F., Kurth, Mark, Lukacs, Gergely, Veit, G., Veit, Guido, Beekman, Jeffrey M., Hegedűs, Tamás, Soya, Naoto, Bagdany, M., Lukacs, Gergely L., Dekkers, Johanna, Xu, Haijin, Okiyoneda, Tsukasa, Verkman, Alan S., Simon, Ágnes, Roldan, Ariel, Dekkers, Johanna F.   +21 more
core   +1 more source

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. Most cystic fibrosis patients have at least one copy of the F508del CFTR mutation, which results in a protein retained in the endoplasmic reticulum and degraded by the ...
Carla M. P. Ribeiro, Martina Gentzsch
openaire   +3 more sources

Efficacy and Safety of Elexacaftor-Tezacaftor-Ivacaftor in the Treatment of Cystic Fibrosis: A Systematic Review

Children, 2023
Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) is a new CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) modulator treatment, used over the last few years, which has shown an improvement in different clinical outcomes in patients with cystic ...
Nikoletta Kapouni, Maria Moustaki, Konstantinos Douros, Ioanna Loukou   +3 more
doaj   +1 more source