Novel Golden Lipid Nanoparticles with Small Interference Ribonucleic Acid for Substrate Reduction Therapy in Fabry Disease
dc.contributor.author | Beraza Millor, Marina | |
dc.contributor.author | Rodríguez Castejón, Julen | |
dc.contributor.author | Miranda Gómez, Jonatan | |
dc.contributor.author | Del Pozo Rodríguez, Ana | |
dc.contributor.author | Rodríguez Gascón, Alicia | |
dc.contributor.author | Solinís Aspiazu, María Ángeles | |
dc.date.accessioned | 2023-08-07T10:24:22Z | |
dc.date.available | 2023-08-07T10:24:22Z | |
dc.date.issued | 2023-07-12 | |
dc.identifier.citation | Pharmaceutics 15(7) : (2023) // Article ID 1936 | es_ES |
dc.identifier.issn | 1999-4923 | |
dc.identifier.uri | http://hdl.handle.net/10810/62115 | |
dc.description.abstract | Substrate reduction therapy (SRT) has been proposed as a new gene therapy for Fabry disease (FD) to prevent the formation of globotriaosylceramide (Gb3). Nanomedicines containing different siRNA targeted to Gb3 synthase (Gb3S) were designed. Formulation factors, such as the composition, solid lipid nanoparticles (SLNs) preparation method and the incorporation of different ligands, such as gold nanoparticles (GNs), protamine (P) and polysaccharides, were evaluated. The new siRNA–golden LNPs were efficiently internalized in an FD cell model (IMFE-1), with GNs detected in the cytoplasm and in the nucleus. Silencing efficacy (measured by RT-qPCR) depended on the final composition and method of preparation, with silencing rates up to 90% (expressed as the reduction in Gb3S-mRNA). GNs conferred a higher system efficacy and stability without compromising cell viability and hemocompatibility. Immunocytochemistry assays confirmed Gb3S silencing for at least 15 days with the most effective formulations. Overall, these results highlight the potential of the new siRNA–golden LNP system as a promising nanomedicine to address FD by specific SRT. | es_ES |
dc.description.sponsorship | This research was funded by MCIU/AEI/FEDER, UE, grant number RTI2018-098672-B-I00; by the UNIVERSITY OF THE BASQUE COUNTRY UPV/EHU, grant number GIU20/048; and by the BASQUE GOVERNMENT, grant number IT1587-22, GIC21/34. | es_ES |
dc.language.iso | eng | es_ES |
dc.publisher | MDPI | es_ES |
dc.relation | info:eu-repo/grantAgreement/MICIU/RTI2018-098672-B-I00 | es_ES |
dc.rights | info:eu-repo/semantics/openAccess | es_ES |
dc.rights.uri | http://creativecommons.org/licenses/by/4.0/ | |
dc.subject | substrate reduction therapy | es_ES |
dc.subject | solid lipid nanoparticles | es_ES |
dc.subject | siRNA | es_ES |
dc.subject | Fabry disease | es_ES |
dc.subject | Gb3 synthase | es_ES |
dc.subject | gold nanoparticles | es_ES |
dc.title | Novel Golden Lipid Nanoparticles with Small Interference Ribonucleic Acid for Substrate Reduction Therapy in Fabry Disease | es_ES |
dc.type | info:eu-repo/semantics/article | es_ES |
dc.date.updated | 2023-07-28T12:21:58Z | |
dc.rights.holder | © 2023 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/ 4.0/). | es_ES |
dc.relation.publisherversion | https://www.mdpi.com/1999-4923/15/7/1936 | es_ES |
dc.identifier.doi | 10.3390/pharmaceutics15071936 | |
dc.departamentoes | Farmacia y ciencias de los alimentos | |
dc.departamentoeu | Farmazia eta elikagaien zientziak |
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Except where otherwise noted, this item's license is described as © 2023 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/ 4.0/).