iPSC-Derived Intestinal Organoids from Cystic Fibrosis Patients Acquire CFTR Activity upon TALEN-Mediated Repair of the p.F508del Mutation

iPSC-Derived Intestinal Organoids from Cystic Fibrosis Patients Acquire CFTR Activity upon TALEN-Mediated Repair of the p.F508del Mutation

Cystic fibrosis (CF) is the main genetic cause of death among the Caucasian population. The disease is characterized by abnormal fluid and electrolyte mobility across secretory epithelia. The first manifestations occur within hours of birth (meconium ileus), later extending to other organs, generall...

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Bibliographic Details
Main Authors: Aarne Fleischer, Sara Vallejo-Díez, José María Martín-Fernández, Almudena Sánchez-Gilabert, Mónica Castresana, Angel del Pozo, Amaia Esquisabel, Silvia Ávila, José Luis Castrillo, Eusebio Gaínza, José Luis Pedraz, Miguel Viñas, Daniel Bachiller
Format: Article
Language:English
Published: Elsevier 2020-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
iPSC
Cystic Fibrosis
gene targeting
TALEN
intestinal organoids
FIS assay
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050120300656

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http://www.sciencedirect.com/science/article/pii/S2329050120300656

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