Por favor, use este identificador para citar o enlazar a este item:
http://hdl.handle.net/10261/221079
COMPARTIR / EXPORTAR:
SHARE CORE BASE | |
Visualizar otros formatos: MARC | Dublin Core | RDF | ORE | MODS | METS | DIDL | DATACITE | |
Título: | Non-viral mediated gene therapy in human cystic fibrosis airway epithelial cells recovers chloride channel functionality |
Autor: | Sainz-Ramos, Myriam; Villate-Beitia, Ilia; Gallego, Idoia; Qtaish, Nuseibah A. L.; Lopez-Mendez, Tania B.; Eritja Casadellà, Ramón CSIC ORCID ; Grijalvo, Santiago CSIC ORCID; Puras, Gustavo; Pedraz, José Luís | Palabras clave: | Gene therapy Non-viral Niosomes CFTR Cystic fibrosis |
Fecha de publicación: | 15-oct-2020 | Editor: | Elsevier | Citación: | International Journal of Pharmaceutics 588: 119757 (2020) | Resumen: | Gene therapy strategies based on non-viral vectors are currently considered as a promising therapeutic option for the treatment of cystic fibrosis (CF), being liposomes the most commonly used gene carriers. Niosomes offer a powerful alternative to liposomes due to their higher stability and lower cytotoxicity, provided by their non-ionic surfactant and helper components. In this work, a three-formulation screening is performed, in terms of physicochemical and biological behavior, in CF patient derived airway epithelial cells. The most efficient niosome formulation reaches 28% of EGFP expressing live cells and follows caveolae-mediated endocytosis. Transfection with therapeutic cystic fibrosis transmembrane conductance regulator (CFTR) gene results in 5-fold increase of CFTR protein expression in transfected versus non-transfected cells, which leads to 1.5-fold increment of the chloride channel functionality. These findings highlight the relevance of niosome-based systems as an encouraging non-viral gene therapy platform with potential therapeutic benefits for CF. | Versión del editor: | https://doi.org/10.1016/j.ijpharm.2020.119757 | URI: | http://hdl.handle.net/10261/221079 | DOI: | 10.1016/j.ijpharm.2020.119757 |
Aparece en las colecciones: | (IQAC) Artículos |
Ficheros en este ítem:
Fichero | Descripción | Tamaño | Formato | |
---|---|---|---|---|
SainzRamos2020IntJPharm.pdf | Artículo principal | 1,15 MB | Adobe PDF | Visualizar/Abrir |
CORE Recommender
SCOPUSTM
Citations
13
checked on 08-abr-2024
WEB OF SCIENCETM
Citations
12
checked on 25-feb-2024
Page view(s)
86
checked on 23-abr-2024
Download(s)
255
checked on 23-abr-2024
Google ScholarTM
Check
Altmetric
Altmetric
NOTA: Los ítems de Digital.CSIC están protegidos por copyright, con todos los derechos reservados, a menos que se indique lo contrario.