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

BACKGROUND: The best investigational drug to treat cystic fibrosis (CF) patients with the most common CF-causing mutation (F508del) is VX-809 (lumacaftor) which recently succeeded in Phase III clinical trial in combination with ivacaftor. This corrector rescues F508del-CFTR from its abnormal intrace...

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Publicat a:EBioMedicine
Autors principals: Awatade, Nikhil T., Uliyakina, Inna, Farinha, Carlos M., Clarke, Luka A., Mendes, Karina, Solé, Amparo, Pastor, Juan, Ramos, Maria Margarida, Amaral, Margarida D.
Format: Artigo
Idioma:Inglês
Publicat: Elsevier 2014
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Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC4484512/
https://ncbi.nlm.nih.gov/pubmed/26137539
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ebiom.2014.12.005
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