Measurements of Functional Responses in Human Primary Lung Cells as a Basis for Personalized Therapy for Cystic Fibrosis

Awatade, Nikhil T.; Uliyakina, Inna; Farinha, Carlos M.; Clarke, Luka A.; Mendes, Karina; Solé, Amparo; Pastor, Juan; Ramos, Maria Margarida; Amaral, Margarida D.

doi:10.1016/j.ebiom.2014.12.005

Measurements of Functional Responses in Human Primary Lung Cells as a Basis for Personalized Therapy for Cystic Fibrosis

Journal Article · Sat Jan 31 23:00:00 EST 2015 · EBioMedicine

DOI:https://doi.org/10.1016/j.ebiom.2014.12.005· OSTI ID:2279389

Awatade, Nikhil T.; Uliyakina, Inna; Farinha, Carlos M.; Clarke, Luka A.; Mendes, Karina; Solé, Amparo; Pastor, Juan; ;

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Sponsoring Organization:: USDOE

OSTI ID:: 2279389

Journal Information:: EBioMedicine, Journal Name: EBioMedicine Journal Issue: 2 Vol. 2; ISSN 2352-3964

Publisher:: ElsevierCopyright Statement

Country of Publication:: Netherlands

Language:: English

References (20)

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Revertants, Low Temperature, and Correctors Reveal the Mechanism of F508del-CFTR Rescue by VX-809 and Suggest Multiple Agents for Full Correction Farinha, Carlos M.; King-Underwood, John; Sousa, Marisa Chemistry & Biology, Vol. 20, Issue 7 https://doi.org/10.1016/j.chembiol.2013.06.004	journal	July 2013
VX-809 and Related Corrector Compounds Exhibit Secondary Activity Stabilizing Active F508del-CFTR after Its Partial Rescue to the Cell Surface Eckford, Paul D. W.; Ramjeesingh, Mohabir; Molinski, Steven Chemistry & Biology, Vol. 21, Issue 5 https://doi.org/10.1016/j.chembiol.2014.02.021	journal	May 2014
The prevalence of cystic fibrosis in the European Union Farrell, Philip M. Journal of Cystic Fibrosis, Vol. 7, Issue 5 https://doi.org/10.1016/j.jcf.2008.03.007	journal	September 2008
Effect of ivacaftor on CFTR forms with missense mutations associated with defects in protein processing or function Van Goor, Fredrick; Yu, Haihui; Burton, Bill Journal of Cystic Fibrosis, Vol. 13, Issue 1 https://doi.org/10.1016/j.jcf.2013.06.008	journal	January 2014
New pharmacological approaches for cystic fibrosis: Promises, progress, pitfalls Bell, Scott C.; De Boeck, Kris; Amaral, Margarida D. Pharmacology & Therapeutics, Vol. 145 https://doi.org/10.1016/j.pharmthera.2014.06.005	journal	January 2015
HGF Stimulation of Rac1 Signaling Enhances Pharmacological Correction of the Most Prevalent Cystic Fibrosis Mutant F508del-CFTR Moniz, Sónia; Sousa, Marisa; Moraes, Bruno José ACS Chemical Biology, Vol. 8, Issue 2 https://doi.org/10.1021/cb300484r	journal	November 2012
Defining the disease liability of variants in the cystic fibrosis transmembrane conductance regulator gene Sosnay, Patrick R.; Siklosi, Karen R.; Van Goor, Fredrick Nature Genetics, Vol. 45, Issue 10 https://doi.org/10.1038/ng.2745	journal	August 2013
A CFTR Potentiator in Patients with Cystic Fibrosis and the G551D Mutation Ramsey, Bonnie W.; Davies, Jane; McElvaney, N. Gerard New England Journal of Medicine, Vol. 365, Issue 18 https://doi.org/10.1056/NEJMoa1105185	journal	November 2011
Revertant mutants G550E and 4RK rescue cystic fibrosis mutants in the first nucleotide-binding domain of CFTR by different mechanisms Roxo-Rosa, Mónica; Xu, Zhe; Schmidt, André Proceedings of the National Academy of Sciences, Vol. 103, Issue 47 https://doi.org/10.1073/pnas.0608312103	journal	November 2006
Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809 Van Goor, F.; Hadida, S.; Grootenhuis, P. D. J. Proceedings of the National Academy of Sciences, Vol. 108, Issue 46 https://doi.org/10.1073/pnas.1105787108	journal	October 2011
Correctors of ΔF508 CFTR restore global conformational maturation without thermally stabilizing the mutant protein He, Lihua; Kota, Pradeep; Aleksandrov, Andrei A. The FASEB Journal, Vol. 27, Issue 2 https://doi.org/10.1096/fj.12-216119	journal	October 2012
CFTR potentiators partially restore channel function to A561E‐CFTR, a cystic fibrosis mutant with a similar mechanism of dysfunction as F508del‐CFTR Wang, Yiting; Liu, Jia; Loizidou, Avgi British Journal of Pharmacology, Vol. 171, Issue 19 https://doi.org/10.1111/bph.12791	journal	September 2014
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