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

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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Bibliographic Details
Main Authors: Nikhil T. Awatade, Inna Uliyakina, Carlos M. Farinha, Luka A. Clarke, Karina Mendes, Amparo Solé, Juan Pastor, Maria Margarida Ramos, Margarida D. Amaral
Format: Article
Language:English
Published: Elsevier 2015-02-01
Series:EBioMedicine
Subjects:
Innovative treatments
Mutation-specific therapies
Personalized medicine
Rare diseases
Online Access:http://www.sciencedirect.com/science/article/pii/S2352396414000577

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http://www.sciencedirect.com/science/article/pii/S2352396414000577

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