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dc.contributor.authorRodríguez Castejón, Julen
dc.contributor.authorBeraza Millor, Marina ORCID
dc.contributor.authorSolinís Aspiazu, María Ángeles ORCID
dc.contributor.authorRodríguez Gascón, Alicia
dc.contributor.authorDel Pozo Rodríguez, Ana ORCID
dc.date.accessioned2024-09-20T15:40:15Z
dc.date.available2024-09-20T15:40:15Z
dc.date.issued2024-10
dc.identifier.citationDrug Delivery and Translational Research 14(10) : 2615-2628 (2024)es_ES
dc.identifier.issn2190-393X
dc.identifier.issn2190-3948
dc.identifier.urihttp://hdl.handle.net/10810/69498
dc.description.abstractFabry disease (FD) results from a lack of activity of the lysosomal enzyme α-Galactosidase A (α-Gal A), leading to the accumulation of glycosphingolipids in several different cell types. Protein supplementation by pDNA or mRNA delivery presents a promising strategy to tackle the underlying genetic defect in FD. Protein-coding nucleic acids in FD can be either delivered to the most affected sites by the disease, including heart, kidney and brain, or to specialized organs that can act as a production factory of the enzyme, such as the liver. Lipid-based systems are currently at the top of the ranking of non-viral nucleic acid delivery systems, and their versatility allows the linking to the surface of a wide range of molecules to control their biodistribution after intravenous administration. This systematic review follows the Preferred Reporting Items for Systematic Reviews and Meta-Analysis (PRISMA) statement guidelines and provides an overview and discussion of the targeting ligands that have been employed so far to actively vectorize intravenously administered non-viral vectors based on lipid carriers to clinically relevant organs in the treatment of FD, for protein-coding nucleic acid (pDNA and mRNA) supplementation. Among the thirty-two studies included, the majority focus on targeting the liver and brain. The targeting of the heart has been reported to a lesser degree, whereas no articles addressing kidney-targeting have been recorded. Although a great effort has been made to develop organ-specific nucleic acid delivery systems, the design of active-targeted carriers with high quality, good clinical translation, and large-scale manufacturing capacity is still challenging.es_ES
dc.description.sponsorshipThis 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. Open Access funding provided thanks to the CRUE-CSIC agreement with Springer Nature.es_ES
dc.language.isoenges_ES
dc.publisherSpringer Naturees_ES
dc.rightsinfo:eu-repo/semantics/openAccesses_ES
dc.rights.urihttp://creativecommons.org/licenses/by/3.0/es/*
dc.subjectFabry diseasees_ES
dc.subjecttargetinges_ES
dc.subjectnucleic acides_ES
dc.subjectlipid nanoparticleses_ES
dc.subjectPRISMAes_ES
dc.titleTargeting strategies with lipid vectors for nucleic acid supplementation therapy in Fabry disease: a systematic reviewes_ES
dc.typeinfo:eu-repo/semantics/articlees_ES
dc.rights.holder© The Author(s) 2024. This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article’s Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons. org/licenses/by/4.0/.es_ES
dc.rights.holderAtribución 3.0 España*
dc.relation.publisherversionhttps://link.springer.com/article/10.1007/s13346-024-01583-0es_ES
dc.identifier.doi10.1007/s13346-024-01583-0
dc.departamentoesFarmacia y ciencias de los alimentoses_ES
dc.departamentoeuFarmazia eta elikagaien zientziakes_ES


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© The Author(s) 2024. This article is licensed under a Creative Commons
Attribution 4.0 International License, which permits use, sharing,
adaptation, distribution and reproduction in any medium or format,
as long as you give appropriate credit to the original author(s) and the
source, provide a link to the Creative Commons licence, and indicate
if changes were made. The images or other third party material in this
article are included in the article’s Creative Commons licence, unless
indicated otherwise in a credit line to the material. If material is not
included in the article’s Creative Commons licence and your intended
use is not permitted by statutory regulation or exceeds the permitted
use, you will need to obtain permission directly from the copyright
holder. To view a copy of this licence, visit http://creativecommons.
org/licenses/by/4.0/.
Except where otherwise noted, this item's license is described as © The Author(s) 2024. This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article’s Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons. org/licenses/by/4.0/.