CFTR functional assays in drug development
作者： Sara M. Zak , John P. Clancy , John J. Brewington
DOI: 10.1080/21678707.2017.1393413
关键词:
摘要: ABSTRACTIntroduction: Cystic Fibrosis (CF) is the most common fatal genetic disease in persons of Northern European ancestry. It an autosomal recessive caused by loss function ...
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Steven M. Rowe, John Paul Clancy, Michael Wilschanski, Nasal Potential Difference Measurements to Assess CFTR Ion Channel Activity Methods in Molecular Biology. ,vol. 741, pp. 69- 86 ,(2011) , 10.1007/978-1-61779-117-8_6
Timothy Neuberger, Bill Burton, Heather Clark, Fredrick Van Goor, Use of Primary Cultures of Human Bronchial Epithelial Cells Isolated from Cystic Fibrosis Patients for the Pre-clinical Testing of CFTR Modulators Methods in Molecular Biology. ,vol. 741, pp. 39- 54 ,(2011) , 10.1007/978-1-61779-117-8_4
R. Boucher, Regulation of airway surface liquid volume by human airway epithelia Pflügers Archiv: European Journal of Physiology. ,vol. 445, pp. 495- 498 ,(2003) , 10.1007/S00424-002-0955-1
Nicoletta Pedemonte, Olga Zegarra-Moran, Luis J.V. Galietta, High-Throughput Screening of Libraries of Compounds to Identify CFTR Modulators Methods in Molecular Biology. ,vol. 741, pp. 13- 21 ,(2011) , 10.1007/978-1-61779-117-8_2
Kavitha Kotha, Rhonda D. Szczesniak, Anjaparavanda P. Naren, Matthew C. Fenchel, Leo L. Duan, Gary L. McPhail, John P. Clancy, Concentration of fractional excretion of nitric oxide (FENO): A potential airway biomarker of restored CFTR function. Journal of Cystic Fibrosis. ,vol. 14, pp. 733- 740 ,(2015) , 10.1016/J.JCF.2015.07.002
D.D. Zomer-van Ommen, L.A.W. Vijftigschild, E. Kruisselbrink, A.M. Vonk, J.F. Dekkers, H.M. Janssens, K.M. de Winter-de Groot, C.K. van der Ent, J.M. Beekman, Limited premature termination codon suppression by read-through agents in cystic fibrosis intestinal organoids Journal of Cystic Fibrosis. ,vol. 15, pp. 158- 162 ,(2016) , 10.1016/J.JCF.2015.07.007
Claire E Wainwright, J Stuart Elborn, Bonnie W Ramsey, Gautham Marigowda, Xiaohong Huang, Marco Cipolli, Carla Colombo, Jane C Davies, Kris De Boeck, Patrick A Flume, Michael W Konstan, Susanna A McColley, Karen McCoy, Edward F McKone, Anne Munck, Felix Ratjen, Steven M Rowe, David Waltz, Michael P Boyle, None, Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR The New England Journal of Medicine. ,vol. 373, pp. 220- 231 ,(2015) , 10.1056/NEJMOA1409547
Dragana Vidović, Marianne S. Carlon, Mélanie F. da Cunha, Johanna F. Dekkers, Monika I. Hollenhorst, Marcel J. C. Bijvelds, Anabela S. Ramalho, Chris Van den Haute, Marc Ferrante, Veerle Baekelandt, Hettie M. Janssens, Kris De Boeck, Isabelle Sermet-Gaudelus, Hugo R. de Jonge, Rik Gijsbers, Jeffrey M. Beekman, Aleksander Edelman, Zeger Debyser, rAAV-CFTRΔR Rescues the Cystic Fibrosis Phenotype in Human Intestinal Organoids and Cystic Fibrosis Mice American Journal of Respiratory and Critical Care Medicine. ,vol. 193, pp. 288- 298 ,(2016) , 10.1164/RCCM.201505-0914OC
Lewis E. Gibson, Robert E. Cooke, A TEST FOR CONCENTRATION OF ELECTROLYTES IN SWEAT IN CYSTIC FIBROSIS OF THE PANCREAS UTILIZING PILOCARPINE BY IONTOPHORESIS Pediatrics. ,vol. 23, pp. 545- 549 ,(1959)
John P. Clancy, Steven M. Rowe, Zsuzsa Bebok, Moira L. Aitken, Ron Gibson, Pam Zeitlin, Pierre Berclaz, Rick Moss, Michael R. Knowles, Robert A. Oster, Nicole Mayer-Hamblett, Bonnie Ramsey, No Detectable Improvements in Cystic Fibrosis Transmembrane Conductance Regulator by Nasal Aminoglycosides in Patients with Cystic Fibrosis with Stop Mutations American Journal of Respiratory Cell and Molecular Biology. ,vol. 37, pp. 57- 66 ,(2007) , 10.1165/RCMB.2006-0173OC