Improved synthesis and characterization of cholesteryl oleate-loaded cationic solid lipid nanoparticles with high transfection efficiency for gene therapy applications

The development of new nanoparticle formulations that are capable of high transfection efficiency without toxicity is essential to provide new tools for gene therapy. However, the issues of complex, poorly reproducible manufacturing methods, and low efficiencies during in vivo testing have prevented...

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Autores: Suñé Pou, Marc, Limeres, Maria J., Nofrerias Roig, Isaac, Nardi Ricart, Anna, Prieto-Sánchez, Silvia, El Yousfi, Younes, Pérez Lozano, Pilar, García Montoya, Encarna, Miñarro Carmona, Montserrat, Ticó Grau, Josep R., Hernández-Munain, Cristina, Suñé, Carlos, Suñé i Negre, Josep M. (Josep Maria)
Tipo de recurso: artículo
Estado:Versión aceptada para publicación
Fecha de publicación:2019
País:España
Institución:Universidad de Barcelona
Repositorio:Dipòsit Digital de la UB
OAI Identifier:oai:diposit.ub.edu:2445/169196
Acceso en línea:https://hdl.handle.net/2445/169196
Access Level:acceso abierto
Palabra clave:Nanopartícules
Lípids
Teràpia genètica
Nanoparticles
Lipids
Gene therapy
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spelling Improved synthesis and characterization of cholesteryl oleate-loaded cationic solid lipid nanoparticles with high transfection efficiency for gene therapy applicationsSuñé Pou, MarcLimeres, Maria J.Nofrerias Roig, IsaacNardi Ricart, AnnaPrieto-Sánchez, SilviaEl Yousfi, YounesPérez Lozano, PilarGarcía Montoya, EncarnaMiñarro Carmona, MontserratTicó Grau, Josep R.Hernández-Munain, CristinaSuñé, CarlosSuñé i Negre, Josep M. (Josep Maria)NanopartículesLípidsTeràpia genèticaNanoparticlesLipidsGene therapyThe development of new nanoparticle formulations that are capable of high transfection efficiency without toxicity is essential to provide new tools for gene therapy. However, the issues of complex, poorly reproducible manufacturing methods, and low efficiencies during in vivo testing have prevented translation to the clinic. We have previously reported the use of cholesteryl oleate as a novel excipient for solid lipid nanoparticles (SLNs) for the development of highly efficient and nontoxic nucleic acid delivery carriers. Here, we performed an extensive characterization of this novel formulation to make the scale up under Good Manufacturing Practice (GMP) possible. We also describe the complete physicochemical and biological characterization of cholesteryl oleate-loaded SLNs to ensure the reproducibility of this formula and the preservation of its characteristics before and after the lyophilization process. We defined the best manufacturing method and studied the influence of some parameters on the obtained nanoparticles using the Quality by Design (ICH Q8) guideline to obtain cholesteryl oleate-loaded SLNs that remain stable during storage and guarantee in vitro nucleic acid delivery efficacy. Our results indicate that this improved formulation is suitable for gene therapy with the possibility of scale-up the manufacturing of nanoparticles under GMP conditions.Elsevier B.V.2019info:eu-repo/semantics/articleinfo:eu-repo/semantics/acceptedVersionapplication/pdfhttps://hdl.handle.net/2445/169196Articles publicats en revistes (Farmàcia, Tecnologia Farmacèutica i Fisicoquímica)reponame:Dipòsit Digital de la UBinstname:Universidad de BarcelonaInglésVersió postprint del document publicat a: https://doi.org/10.1016/j.colsurfb.2019.04.037Colloids and Surfaces B-Biointerfaces, 2019, vol. 180, p. 159-167https://doi.org/10.1016/j.colsurfb.2019.04.037cc-by-nc-nd (c) Elsevier B.V., 2019http://creativecommons.org/licenses/by-nc-nd/3.0/esinfo:eu-repo/semantics/openAccessoai:diposit.ub.edu:2445/1691962026-05-27T06:46:51Z
dc.title.none.fl_str_mv Improved synthesis and characterization of cholesteryl oleate-loaded cationic solid lipid nanoparticles with high transfection efficiency for gene therapy applications
title Improved synthesis and characterization of cholesteryl oleate-loaded cationic solid lipid nanoparticles with high transfection efficiency for gene therapy applications
spellingShingle Improved synthesis and characterization of cholesteryl oleate-loaded cationic solid lipid nanoparticles with high transfection efficiency for gene therapy applications
Suñé Pou, Marc
Nanopartícules
Lípids
Teràpia genètica
Nanoparticles
Lipids
Gene therapy
title_short Improved synthesis and characterization of cholesteryl oleate-loaded cationic solid lipid nanoparticles with high transfection efficiency for gene therapy applications
title_full Improved synthesis and characterization of cholesteryl oleate-loaded cationic solid lipid nanoparticles with high transfection efficiency for gene therapy applications
title_fullStr Improved synthesis and characterization of cholesteryl oleate-loaded cationic solid lipid nanoparticles with high transfection efficiency for gene therapy applications
title_full_unstemmed Improved synthesis and characterization of cholesteryl oleate-loaded cationic solid lipid nanoparticles with high transfection efficiency for gene therapy applications
title_sort Improved synthesis and characterization of cholesteryl oleate-loaded cationic solid lipid nanoparticles with high transfection efficiency for gene therapy applications
dc.creator.none.fl_str_mv Suñé Pou, Marc
Limeres, Maria J.
Nofrerias Roig, Isaac
Nardi Ricart, Anna
Prieto-Sánchez, Silvia
El Yousfi, Younes
Pérez Lozano, Pilar
García Montoya, Encarna
Miñarro Carmona, Montserrat
Ticó Grau, Josep R.
Hernández-Munain, Cristina
Suñé, Carlos
Suñé i Negre, Josep M. (Josep Maria)
author Suñé Pou, Marc
author_facet Suñé Pou, Marc
Limeres, Maria J.
Nofrerias Roig, Isaac
Nardi Ricart, Anna
Prieto-Sánchez, Silvia
El Yousfi, Younes
Pérez Lozano, Pilar
García Montoya, Encarna
Miñarro Carmona, Montserrat
Ticó Grau, Josep R.
Hernández-Munain, Cristina
Suñé, Carlos
Suñé i Negre, Josep M. (Josep Maria)
author_role author
author2 Limeres, Maria J.
Nofrerias Roig, Isaac
Nardi Ricart, Anna
Prieto-Sánchez, Silvia
El Yousfi, Younes
Pérez Lozano, Pilar
García Montoya, Encarna
Miñarro Carmona, Montserrat
Ticó Grau, Josep R.
Hernández-Munain, Cristina
Suñé, Carlos
Suñé i Negre, Josep M. (Josep Maria)
author2_role author
author
author
author
author
author
author
author
author
author
author
author
dc.subject.none.fl_str_mv Nanopartícules
Lípids
Teràpia genètica
Nanoparticles
Lipids
Gene therapy
topic Nanopartícules
Lípids
Teràpia genètica
Nanoparticles
Lipids
Gene therapy
description The development of new nanoparticle formulations that are capable of high transfection efficiency without toxicity is essential to provide new tools for gene therapy. However, the issues of complex, poorly reproducible manufacturing methods, and low efficiencies during in vivo testing have prevented translation to the clinic. We have previously reported the use of cholesteryl oleate as a novel excipient for solid lipid nanoparticles (SLNs) for the development of highly efficient and nontoxic nucleic acid delivery carriers. Here, we performed an extensive characterization of this novel formulation to make the scale up under Good Manufacturing Practice (GMP) possible. We also describe the complete physicochemical and biological characterization of cholesteryl oleate-loaded SLNs to ensure the reproducibility of this formula and the preservation of its characteristics before and after the lyophilization process. We defined the best manufacturing method and studied the influence of some parameters on the obtained nanoparticles using the Quality by Design (ICH Q8) guideline to obtain cholesteryl oleate-loaded SLNs that remain stable during storage and guarantee in vitro nucleic acid delivery efficacy. Our results indicate that this improved formulation is suitable for gene therapy with the possibility of scale-up the manufacturing of nanoparticles under GMP conditions.
publishDate 2019
dc.date.none.fl_str_mv 2019
dc.type.none.fl_str_mv info:eu-repo/semantics/article
info:eu-repo/semantics/acceptedVersion
format article
status_str acceptedVersion
dc.identifier.none.fl_str_mv https://hdl.handle.net/2445/169196
url https://hdl.handle.net/2445/169196
dc.language.none.fl_str_mv Inglés
language_invalid_str_mv Inglés
dc.relation.none.fl_str_mv Versió postprint del document publicat a: https://doi.org/10.1016/j.colsurfb.2019.04.037
Colloids and Surfaces B-Biointerfaces, 2019, vol. 180, p. 159-167
https://doi.org/10.1016/j.colsurfb.2019.04.037
dc.rights.none.fl_str_mv cc-by-nc-nd (c) Elsevier B.V., 2019
http://creativecommons.org/licenses/by-nc-nd/3.0/es
info:eu-repo/semantics/openAccess
rights_invalid_str_mv cc-by-nc-nd (c) Elsevier B.V., 2019
http://creativecommons.org/licenses/by-nc-nd/3.0/es
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv application/pdf
dc.publisher.none.fl_str_mv Elsevier B.V.
publisher.none.fl_str_mv Elsevier B.V.
dc.source.none.fl_str_mv Articles publicats en revistes (Farmàcia, Tecnologia Farmacèutica i Fisicoquímica)
reponame:Dipòsit Digital de la UB
instname:Universidad de Barcelona
instname_str Universidad de Barcelona
reponame_str Dipòsit Digital de la UB
collection Dipòsit Digital de la UB
repository.name.fl_str_mv
repository.mail.fl_str_mv
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score 15,301603

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