Non-viral vectors based on cationic niosomes and minicircle DNA technology enhance gene delivery efficiency for biomedical applications in retinal disorders

Low transfection efficiency is a major challenge to overcome in non-viral approaches to reach clinical practice. Our aim was to explore new strategies to achieve more efficient non-viral gene therapies for clinical applications and in particular, for retinal diseases. Cationic niosomes and three GFP...

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Detalhes bibliográficos
Autores: Gallego , Idoia, Villate Beitia, Ilia, Martínez-Navarrete, Gema, Menéndez, Margarita, López-Méndez, Tania, Soto-Sánchez, Cristina, Zárate, Jon, Puras, Gustavo, Fernández, Eduardo, Pedraz, José Luis
Formato: artículo
Fecha de publicación:2019
País:España
Recursos:Universidad Miguel Hernández de Elche
Repositorio:REDIUMH. Depósito Digital de la UMH
OAI Identifier:oai:dspace.umh.es:11000/34590
Acesso em linha:https://hdl.handle.net/11000/34590
Access Level:acceso abierto
Palavra-chave:non-viral vector
niosomes
minicircle
transfection
gene therapy
retina
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spelling Non-viral vectors based on cationic niosomes and minicircle DNA technology enhance gene delivery efficiency for biomedical applications in retinal disordersGallego , IdoiaVillate Beitia, IliaMartínez-Navarrete, GemaMenéndez, MargaritaLópez-Méndez, TaniaSoto-Sánchez, CristinaZárate, JonPuras, GustavoFernández, EduardoPedraz, José Luisnon-viral vectorniosomesminicircletransfectiongene therapyretinaLow transfection efficiency is a major challenge to overcome in non-viral approaches to reach clinical practice. Our aim was to explore new strategies to achieve more efficient non-viral gene therapies for clinical applications and in particular, for retinal diseases. Cationic niosomes and three GFP-encoding genetic materials consisting on minicircle (2.3 kb), its parental plasmid (3.5 kb) and a larger plasmid (5.5 kb) were combined to form nioplexes. Once fully physicochemically characterized, in vitro experiments in ARPE-19 retina epithelial cells showed that transfection efficiency of minicircle nioplexes doubled that of plasmids ones, maintaining good cell viability in all cases. Transfections in retinal primary cells and injections of nioplexes in rat retinas confirmed the higher capacity of cationic niosomes vectoring minicircle to deliver the genetic material into retina cells. Therefore, nioplexes based on cationic niosomes vectoring minicircle DNA represent a potential tool for the treatment of inherited retinal diseases.ElsevierDepartamentos de la UMH::Histología y Anatomía202520252019info:eu-repo/semantics/articleapplication/pdf11application/pdfhttps://hdl.handle.net/11000/34590reponame:REDIUMH. Depósito Digital de la UMHinstname:Universidad Miguel Hernández de ElcheInglés10.1016/j.nano.2018.12.018info:eu-repo/semantics/openAccesshttp://creativecommons.org/licenses/by-nc-nd/4.0/oai:dspace.umh.es:11000/345902026-05-27T13:36:21Z
dc.title.none.fl_str_mv Non-viral vectors based on cationic niosomes and minicircle DNA technology enhance gene delivery efficiency for biomedical applications in retinal disorders
title Non-viral vectors based on cationic niosomes and minicircle DNA technology enhance gene delivery efficiency for biomedical applications in retinal disorders
spellingShingle Non-viral vectors based on cationic niosomes and minicircle DNA technology enhance gene delivery efficiency for biomedical applications in retinal disorders
Gallego , Idoia
non-viral vector
niosomes
minicircle
transfection
gene therapy
retina
title_short Non-viral vectors based on cationic niosomes and minicircle DNA technology enhance gene delivery efficiency for biomedical applications in retinal disorders
title_full Non-viral vectors based on cationic niosomes and minicircle DNA technology enhance gene delivery efficiency for biomedical applications in retinal disorders
title_fullStr Non-viral vectors based on cationic niosomes and minicircle DNA technology enhance gene delivery efficiency for biomedical applications in retinal disorders
title_full_unstemmed Non-viral vectors based on cationic niosomes and minicircle DNA technology enhance gene delivery efficiency for biomedical applications in retinal disorders
title_sort Non-viral vectors based on cationic niosomes and minicircle DNA technology enhance gene delivery efficiency for biomedical applications in retinal disorders
dc.creator.none.fl_str_mv Gallego , Idoia
Villate Beitia, Ilia
Martínez-Navarrete, Gema
Menéndez, Margarita
López-Méndez, Tania
Soto-Sánchez, Cristina
Zárate, Jon
Puras, Gustavo
Fernández, Eduardo
Pedraz, José Luis
author Gallego , Idoia
author_facet Gallego , Idoia
Villate Beitia, Ilia
Martínez-Navarrete, Gema
Menéndez, Margarita
López-Méndez, Tania
Soto-Sánchez, Cristina
Zárate, Jon
Puras, Gustavo
Fernández, Eduardo
Pedraz, José Luis
author_role author
author2 Villate Beitia, Ilia
Martínez-Navarrete, Gema
Menéndez, Margarita
López-Méndez, Tania
Soto-Sánchez, Cristina
Zárate, Jon
Puras, Gustavo
Fernández, Eduardo
Pedraz, José Luis
author2_role author
author
author
author
author
author
author
author
author
dc.contributor.none.fl_str_mv Departamentos de la UMH::Histología y Anatomía
dc.subject.none.fl_str_mv non-viral vector
niosomes
minicircle
transfection
gene therapy
retina
topic non-viral vector
niosomes
minicircle
transfection
gene therapy
retina
description Low transfection efficiency is a major challenge to overcome in non-viral approaches to reach clinical practice. Our aim was to explore new strategies to achieve more efficient non-viral gene therapies for clinical applications and in particular, for retinal diseases. Cationic niosomes and three GFP-encoding genetic materials consisting on minicircle (2.3 kb), its parental plasmid (3.5 kb) and a larger plasmid (5.5 kb) were combined to form nioplexes. Once fully physicochemically characterized, in vitro experiments in ARPE-19 retina epithelial cells showed that transfection efficiency of minicircle nioplexes doubled that of plasmids ones, maintaining good cell viability in all cases. Transfections in retinal primary cells and injections of nioplexes in rat retinas confirmed the higher capacity of cationic niosomes vectoring minicircle to deliver the genetic material into retina cells. Therefore, nioplexes based on cationic niosomes vectoring minicircle DNA represent a potential tool for the treatment of inherited retinal diseases.
publishDate 2019
dc.date.none.fl_str_mv 2019
2025
2025
dc.type.none.fl_str_mv info:eu-repo/semantics/article
format article
dc.identifier.none.fl_str_mv https://hdl.handle.net/11000/34590
url https://hdl.handle.net/11000/34590
dc.language.none.fl_str_mv Inglés
language_invalid_str_mv Inglés
dc.relation.none.fl_str_mv 10.1016/j.nano.2018.12.018
dc.rights.none.fl_str_mv info:eu-repo/semantics/openAccess
http://creativecommons.org/licenses/by-nc-nd/4.0/
eu_rights_str_mv openAccess
rights_invalid_str_mv http://creativecommons.org/licenses/by-nc-nd/4.0/
dc.format.none.fl_str_mv application/pdf
11
application/pdf
dc.publisher.none.fl_str_mv Elsevier
publisher.none.fl_str_mv Elsevier
dc.source.none.fl_str_mv reponame:REDIUMH. Depósito Digital de la UMH
instname:Universidad Miguel Hernández de Elche
instname_str Universidad Miguel Hernández de Elche
reponame_str REDIUMH. Depósito Digital de la UMH
collection REDIUMH. Depósito Digital de la UMH
repository.name.fl_str_mv
repository.mail.fl_str_mv
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score 15.81155

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