Results 31 to 40 of about 5,785 (196)

New drugs, new challenges in cystic fibrosis care

European Respiratory Review
Cystic fibrosis (CF) is a genetic disease caused by variants in the gene encoding for the CF transmembrane conductance regulator (CFTR) protein, a chloride and bicarbonate channel.
Isabelle Fajac, Pierre-Régis Burgel, Clémence Martin   +2 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

Ligand-based virtual-screening identified a novel CFTR ligand which improves the defective cell surface expression of misfolded ABC transporters

Frontiers in Pharmacology
Cystic fibrosis (CF) is a monogenetic disease caused by the mutation of CFTR, a cAMP-regulated Cl− channel expressing at the apical plasma membrane (PM) of epithelia.
Shogo Taniguchi, Francois Berenger, Yukako Doi, Ayana Mimura, Yoshihiro Yamanishi, Tsukasa Okiyoneda   +5 more
doaj   +1 more source

Small-molecule eRF3a degraders rescue CFTR nonsense mutations by promoting premature termination codon readthrough

The Journal of Clinical Investigation, 2022
The vast majority of people with cystic fibrosis (CF) are now eligible for CF transmembrane regulator (CFTR) modulator therapy. The remaining individuals with CF harbor premature termination codons (PTCs) or rare CFTR variants with limited treatment ...
Rhianna E. Lee, Catherine A. Lewis, Lihua He, Emily C. Bulik-Sullivan, Samuel C. Gallant, Teresa M. Mascenik, Hong Dang, Deborah M. Cholon, Martina Gentzsch, Lisa C. Morton, John T. Minges, Jonathan W. Theile, Neil A. Castle, Michael R. Knowles, Adam J. Kimple, Scott H. Randell   +15 more
doaj   +1 more source

Antisense oligonucleotide targeting the E3 ligase RFFL potentiates CFTR modulator efficacy in CF primary bronchial epithelial cells

Molecular Therapy: Nucleic Acids
Cystic fibrosis (CF) is most commonly caused by the ΔF508 mutation in the CFTR gene, leading to misfolding and degradation of the CFTR protein. Although CFTR modulators such as elexacaftor/tezacaftor/ivacaftor (ETI) provide clinical benefit, their ...
Daichi Hinata, Yukari Kai, Ryosuke Fukuda, Yuka Kamada, Yuuya Kasahara, Kiyomi Sasaki, Tokuyuki Yoshida, Satoshi Obika, Takao Inoue, Tsukasa Okiyoneda   +9 more
doaj   +1 more source

Correction of CFTR function in nasal epithelial cells from cystic fibrosis patients predicts improvement of respiratory function by CFTR modulators

Scientific Reports, 2017
Clinical studies with modulators of the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) protein have demonstrated that functional restoration of the mutated CFTR can lead to substantial clinical benefit.
Iwona M. Pranke, Aurélie Hatton, Juliette Simonin, Jean Philippe Jais, Françoise Le Pimpec-Barthes, Ania Carsin, Pierre Bonnette, Michael Fayon, Nathalie Stremler-Le Bel, Dominique Grenet, Matthieu Thumerel, Julie Mazenq, Valerie Urbach, Myriam Mesbahi, Emanuelle Girodon-Boulandet, Alexandre Hinzpeter, Aleksander Edelman, Isabelle Sermet-Gaudelus   +17 more
doaj   +1 more source

CFTR Modulators: The Changing Face of Cystic Fibrosis in the Era of Precision Medicine

Frontiers in Pharmacology, 2020
Cystic fibrosis (CF) is a lethal inherited disease caused by mutations in the CF transmembrane conductance regulator (CFTR) gene, which result in impairment of CFTR mRNA and protein expression, function, stability or a combination of these.
Miquéias Lopes-Pacheco
doaj   +1 more source

Experience to date with CFTR modulators during pregnancy and breastfeeding in the British Columbia Cystic Fibrosis clinic

Respiratory Medicine Case Reports, 2022
The introduction and rapid uptake of CFTR modulator therapy, in addition to other treatments, has significantly increased life expectancy in CF and provided more women the opportunity to consider and successfully be managed throughout pregnancy. There is
Jodi Goodwin, Bradley S. Quon, Pearce G. Wilcox   +2 more
doaj   +1 more source

CGM patterns in adults with cystic fibrosis-related diabetes before and after elexacaftor-tezacaftor-ivacaftor therapy

Journal of Clinical & Translational Endocrinology, 2022
Cystic fibrosis-related diabetes (CFRD) is a common complication of cystic fibrosis that is associated with worse outcomes and higher mortality rates.
Hanna Crow, Charles Bengtson, Xiaosong Shi, Leland Graves, III, Abeer Anabtawi   +4 more
doaj   +1 more source

Microfluidic Nano‐Assembly of Red‐Blood‐Cell (RBC) Lipids and Components for Engineering Extracellular Vesicles

Advanced Healthcare Materials, EarlyView.
Engineered red blood cell‐derived extracellular vesicles (eRBCEVs) are synthesized via controlled microfluidic assembly from native RBC lipids, enabling tunable encapsulation of proteins, nucleic acids, nanoparticles, and viral vectors. The platform demonstrates reproducible nanoscale architecture, preserved membrane composition, and functional cargo ...
Chiranth K. Nagaraj, Xilal Y. Rima, Kim T. Nguyen, Courtney Culkins, Nana Boateng, Jacob Doon‐Ralls, Alejandro Bresolin, Xin Huang, Hong Li, Vahedi Amid, Ajeet Pal Singh, Dharti Shantaram, Anastasiia Amari, Mangesh D. Hade, Nicholas R. Merriam, Zachary D. Schultz, Scott Q. Harper, Nizar Y. Saad, Setty M. Magaña, Willa A. Hsueh, Rachel E. Kopec, Blaise R. Kimmel, Andre F. Palmer, Eduardo Reátegui   +23 more
wiley   +1 more source