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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Detalles Bibliográficos
Autores principales: Fleischer, Aarne, Vallejo-Díez, Sara, Martín-Fernández, José María, Sánchez-Gilabert, Almudena, Castresana, Mónica, del Pozo, Angel, Esquisabel, Amaia, Ávila, Silvia, Castrillo, José Luis, Gaínza, Eusebio, Pedraz, José Luis, Viñas, Miguel, Bachiller, Daniel
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7195499/
https://www.ncbi.nlm.nih.gov/pubmed/32373648
http://dx.doi.org/10.1016/j.omtm.2020.04.005

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7195499/
https://www.ncbi.nlm.nih.gov/pubmed/32373648
http://dx.doi.org/10.1016/j.omtm.2020.04.005

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