Results 41 to 50 of about 12,467 (245)

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 ...
Michal Shteinberg, Jennifer L. Taylor-Cousar   +1 more
doaj   +1 more source

Current Progress in Therapeutic Gene Editing for Monogenic Diseases [PDF]

, 2016
Programmable nucleases allow defined alterations in the genome with ease-of-use, efficiency, and specificity. Their availability has led to accurate and widespread genome engineering, with multiple applications in basic research, biotechnology, and ...
Moore, Marc, Prakash, Versha, Yáñez-Muñoz, Rafael J   +2 more
core   +1 more source

Beyond the Surface: Novel Therapy Approach for Pancreatitis in the Setting of CFTR Dysfunction. [PDF]

Case Rep Gastrointest Med
Objectives Despite cystic fibrosis transmembrane conductance regulator (CFTR) proteins being present throughout the entire body and organ systems, typical presentation of cystic fibrosis (CF) involves lung disease. We report a series of individuals that were referred to our CF clinic for evaluation following a diagnosis of chronic pancreatitis or acute
Wright BA, Hodapp E, Hornick DB, Peña TA.   +3 more
europepmc   +2 more sources

Restoring Cystic Fibrosis Transmembrane Conductance Regulator Function Reduces Airway Bacteria and Inflammation in People with Cystic Fibrosis and Chronic Lung Infections [PDF]

, 2017
Rationale: Previous work indicates that ivacaftor improves cystic fibrosis transmembrane conductance regulator (CFTR) activity and lung function in people with cystic fibrosis and G551D-CFTR mutations but does not reduce density of bacteria or markers of
Accurso, Frank J., Adam, Ryan J., Bruce, James E., Carter, Suzanne, Cooke, Gordon, Donnelly, Seamas C., Edwards, Rachael M., Gallagher, Charles, Grogan, Brenda, Heltshe, Sonya L., Hisert, Katherine B., Hoffman, Lucas R., Jorth, Peter, Launspach, Jan L., McKone, Edward F., Pope, Christopher, Radey, Matthew, Singh, Pradeep K., Stoltz, David, Welsh, Michael J., Wolter, Daniel J., Wu, Xia   +21 more
core   +2 more sources

Pharmacogenomic testing and its future in community pharmacy [PDF]

, 2020
Although it is common to see pharmacogenomic testing used North America and Australia, it is not yet part of practice in the UK. With the promise of genomic screening becoming part of the NHS, pharmacists must equip themselves with a knowledge of how the
Wright, David, Youssef, Essra
core   +1 more source

Real-world impact of ivacaftor in people with cystic fibrosis and select ivacaftor-responsive mutations

BMJ Open Respiratory Research
Background Ivacaftor approval was extended to people with cystic fibrosis (CF) with ≥1 of 28 additional ivacaftor-responsive mutations in the USA in 2017 based on preclinical in vitro data.
Mark Higgins, Teja Thorat, Craig McKinnon, Alexander Craft   +3 more
doaj   +1 more source

Alteration of Membrane Cholesterol Content Plays a Key Role in Regulation of Cystic Fibrosis Transmembrane Conductance Regulator Channel Activity

Frontiers in Physiology, 2021
Altered cholesterol homeostasis in cystic fibrosis patients has been reported, although controversy remains. As a major membrane lipid component, cholesterol modulates the function of multiple ion channels by complicated mechanisms.
Guiying Cui, Kirsten A. Cottrill, Kerry M. Strickland, Sarah A. Mashburn, Michael Koval, Nael A. McCarty   +5 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

Inhibitory effects of calcium channel blockers nisoldipine and nimodipine on ivacaftor metabolism and their underlying mechanism

Frontiers in Pharmacology
Ivacaftor is the first potentiator of the cystic fibrosis transmembrane conductance regulator (CFTR) protein approved for use alone in the treatment of cystic fibrosis (CF).
Hailun Xia, Xinhao Xu, Jie Chen, Hualu Wu, Yuxin Shen, Xiaohai Chen, Ren-ai Xu, Wenzhi Wu   +7 more
doaj   +1 more source

The impact of ivacaftor on sinonasal pathology in S1251N-mediated cystic fibrosis patients.

PLoS ONE, 2020
ImportanceSinonasal symptoms in patients suffering from cystic fibrosis can negatively influence the quality of life and sinuses can be a niche for pathogens causing infection and inflammation leading to a decrease of lung function.
Romee Gostelie, Inge Stegeman, Gitte Berkers, Joost Bittermann, Ivonne Ligtenberg-van der Drift, Peter-Jan van Kipshagen, Karin de Winter-de Groot, Lucienne Speleman   +7 more
doaj   +1 more source