Results 31 to 40 of about 9,635 (207)

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

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

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

Paediatrics: messages from Munich. [PDF]

, 2015
The aim of this article is to describe paediatric highlights from the 2014 European Respiratory Society (ERS) International Congress in Munich, Germany.
Albuali, Alexopoulos, Andersson, Anton-Pacheco, Arigliani, Arumugam, Balfour-Lynn, Baraldi, Barikbin, Bekhof, Benseler, Berankova, Bilton, Bon, Boon, Boudewyns, Brinkman, Casas, Chiu, Constant, Conway, Dassios, DeBoer, Duijts, Elborn, Engelkes, Engelkes, Everard, Fainardi, Fouzas, Frankenberger, Gadomski, Gilchrist, Goksör, Goussard, Goutaki, Gray, Grosse-Onnebrink, Hatziagorou, Henckel, Hirst, Honková, Iglesias, Iglesias, Ingham, Ioan, Jedynak-Wasowicz, Jung, Kirkby, Kobbernagel, Koschlig, Krivec, Loza, Luciani, Lukkarinen, Madkour, Maguire, Mandadzhieva, Marshall, Maurer, Meurling, Moreno, Moreno, Mulder, Murray, Murray, Mustafayev, Mutti, Naehrig, Ohn, Olsson, Ong, Onland, Parekh, Pie tropaoli, Popovic, Pramana, Ramsey, Rosenow, Rossor, Rubio, Schoegler, Schoegler, Schultz, Serio, Smit, Smyth, Stern, Stokholm, Su, Svedberg, Thouvenin, Tiwari, Umutoglu, Uyan, Van Bragt, Van der Schee, Waddingham, Yacob, Yammine   +99 more
core   +11 more sources

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

Structure of ABCB1/P-Glycoprotein in the Presence of the CFTR Potentiator Ivacaftor

Membranes, 2021
ABCB1/P-glycoprotein is an ATP binding cassette transporter that is involved in the clearance of xenobiotics, and it affects the disposition of many drugs in the body.
Alessandro Barbieri, Nopnithi Thonghin, Talha Shafi, Stephen M. Prince, Richard F. Collins, Robert C. Ford   +5 more
doaj   +1 more source

Some gating potentiators, including VX-770, diminish ΔF508-CFTR functional expression. [PDF]

, 2014
Cystic fibrosis (CF) is caused by mutations in the CF transmembrane regulator (CFTR) that result in reduced anion conductance at the apical membrane of secretory epithelia.
Apaja, Pirjo M., Avramescu, Radu G., Bagdany, Miklos, Borot, Florence, Finkbeiner, Walter E., Hegedus, Tamas, Lukacs, Gergely L., Perdomo, Doranda, Phuan, Puay-Wah, Szollosi, Daniel, Veit, Guido, Verkman, Alan S., Wu, Yu-Sheng   +12 more
core   +1 more source

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

Regulatory T cell enhancement in adults with cystic fibrosis receiving Elexacaftor/Tezacaftor/Ivacaftor therapy

Frontiers in Immunology, 2023
IntroductionCystic fibrosis (CF), especially CF lung disease, is characterized by chronic infection, immune dysfunction including impairment of regulatory T cells (Tregs) and an exaggerated inflammatory response.
Dirk Westhölter, Jonas Raspe, Hendrik Uebner, Johannes Pipping, Mona Schmitz, Svenja Straßburg, Sivagurunathan Sutharsan, Matthias Welsner, Christian Taube, Sebastian Reuter   +9 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