Galactomannan-Decorated Lipidic Nanocarrier for Gene Supplementation Therapy in Fabry Disease
dc.contributor.author | Rodríguez Castejón, Julen | |
dc.contributor.author | Gómez Aguado, Itziar | |
dc.contributor.author | Beraza Millor, Marina | |
dc.contributor.author | Solinís Aspiazu, María Ángeles | |
dc.contributor.author | Del Pozo Rodríguez, Ana | |
dc.contributor.author | Rodríguez Gascón, Alicia | |
dc.date.accessioned | 2022-08-04T12:28:13Z | |
dc.date.available | 2022-08-04T12:28:13Z | |
dc.date.issued | 2022 | |
dc.identifier.citation | Nanomaterials 12(14) : (2022) // Article ID 2339 | es_ES |
dc.identifier.issn | 2079-4991 | |
dc.identifier.uri | http://hdl.handle.net/10810/57207 | |
dc.description.abstract | Gene supplementation therapy with plasmid DNA (pDNA) represents one of the most promising strategies for the treatment of monogenic diseases such as Fabry disease (FD). In the present work, we developed a solid lipid nanoparticles (SLN)-based non-viral vector with a size below 100 nm, and decorated with galactomannan (GM) to target the liver as an α-Galactosidase A (α-Gal A) production factory. After the physicochemical characterization of the GM-SLN vector, cellular uptake, transfection efficacy and capacity to increase α-Gal A activity were evaluated in vitro in a liver cell line (Hep G2) and in vivo in an animal model of FD. The vector showed efficient internalization and it was highly efficient in promoting protein synthesis in Hep G2 cells. Additionally, the vector did not show relevant agglutination of erythrocytes and lacked hemolytic activity. After the systemic administration to Fabry mice, it achieved clinically relevant α-Gal A activity levels in plasma, liver, and other organs, importantly in heart and kidneys, two of the most damaged organs in FD. This work shows the potential application of GM-decorated lipidic nanocarries for the treatment of FD by pDNA-based gene augmentation. | es_ES |
dc.description.sponsorship | This research was funded by MCIU/AEI/FEDER, UE (RTI2018-098672-B-I00) and by the University of the Basque Country UPV/EHU (GIU20/048). | es_ES |
dc.language.iso | eng | es_ES |
dc.publisher | MDPI | es_ES |
dc.relation | info:eu-repo/grantAgreement/MICIU/TI2018-098672-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 | advanced therapies | es_ES |
dc.subject | Fabry disease | es_ES |
dc.subject | galactomannan | es_ES |
dc.subject | gene augmentation | es_ES |
dc.subject | intravenous administration | es_ES |
dc.subject | non-viral vectors | es_ES |
dc.subject | plasmid DNA | es_ES |
dc.subject | solid lipid nanoparticles | es_ES |
dc.subject | targeting; α-galactosidase A | es_ES |
dc.title | Galactomannan-Decorated Lipidic Nanocarrier for Gene Supplementation Therapy in Fabry Disease | es_ES |
dc.type | info:eu-repo/semantics/article | es_ES |
dc.date.updated | 2022-07-25T16:33:46Z | |
dc.rights.holder | © 2022 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/2079-4991/12/14/2339 | es_ES |
dc.identifier.doi | 10.3390/nano12142339 | |
dc.departamentoes | Farmacia y ciencias de los alimentos | |
dc.departamentoeu | Farmazia eta elikagaien zientziak |
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