Results 51 to 60 of about 12,522 (293)

Modulator Therapy for Cystic Fibrosis: An Exploration of Current Research [PDF]

, 2020
Developing a drug therapy that addresses the root cause of cystic fibrosis (CF) by increasing CFTR protein levels has long been a research challenge. After genetic therapy failed because a suitable delivery system could not be found, researchers began ...
Rombocos, Jessalyn
core   +1 more source

CF Fungal Disease in the Age of CFTR Modulators [PDF]

Mycopathologia, 2021
AbstractFungi are increasingly recognised to have a significant role in the progression of lung disease in Cystic fibrosis with Aspergillus fumigatus the most common fungus isolated during respiratory sampling. The emergence of novel CFTR modulators has, however, significantly changed the outlook of disease progression in CF.
Amelia Bercusson, George Jarvis, Anand Shah   +2 more
openaire   +4 more sources

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

Long term in vitro expansion of epithelial stem cells enabled by pharmacological inhibition of PAK1-ROCK-Myosin II and TGF-β signaling [PDF]

, 2018
Summary: Despite substantial self-renewal capability in vivo, epithelial stem and progenitor cells located in various tissues expand for a few passages in vitro in feeder-free condition before they succumb to growth arrest.
Challberg, Sharon S, Lee, Hyung Joo, McQuiston, Travis J, Pollok, Brian A, Shrivastava, Anura, Wang, Ruipeng, Wang, Ting, Zhang, Chengkang   +7 more
core   +2 more sources

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

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

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

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 modulator use and risk of nontuberculous mycobacteria positivity in cystic fibrosis, 2011–2018

ERJ Open Research, 2022
Background People with cystic fibrosis are at increased risk of pulmonary nontuberculous mycobacteria (NTM) disease. Cystic fibrosis transmembrane conductance regulator (CFTR) modulators are associated with reduced lung infection with pathogens like ...
Emily E. Ricotta, D. Rebecca Prevots, Kenneth N. Olivier   +2 more
doaj   +1 more source

CFTR Modulators Therapy Efficacy in Reducing Cystic Fibrosis (CF) Exacerbation and Improving Selected Spirometry Parameters: A Real-Life Study in a Single-Centre Polish Population. [PDF]

J Clin Med
Winiarska HM, Springer D, Wojtaś F, Wysocka E, Cofta S.   +4 more
europepmc   +3 more sources