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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...
Autores principales: | Awatade, Nikhil T., Uliyakina, Inna, Farinha, Carlos M., Clarke, Luka A., Mendes, Karina, Solé, Amparo, Pastor, Juan, Ramos, Maria Margarida, Amaral, Margarida D. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Elsevier
2014
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4484512/ https://www.ncbi.nlm.nih.gov/pubmed/26137539 http://dx.doi.org/10.1016/j.ebiom.2014.12.005 |
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