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Title: Improved synthesis and characterization of cholesteryl oleate-loaded cationic solid lipid nanoparticles with high transfection efficiency for gene therapy applications
Author: 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)
Keywords: Nanopartícules
Teràpia genètica
Gene therapy
Issue Date: 1-Aug-2019
Publisher: Elsevier B.V.
Abstract: 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.
Note: Versió postprint del document publicat a:
It is part of: Colloids and Surfaces B-Biointerfaces, 2019, vol. 180, p. 159-167
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ISSN: 0927-7765
Appears in Collections:Articles publicats en revistes (Farmàcia, Tecnologia Farmacèutica i Fisicoquímica)

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