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dc.contributor.authorÁlvarez González, José Víctor
dc.contributor.authorBravo López, Susana Belén
dc.contributor.authorGarcía Vence, María
dc.contributor.authorCastro López, María José de
dc.contributor.authorLuzardo Álvarez, Asteria María
dc.contributor.authorColón Mejeras, Cristóbal
dc.contributor.authorTomatsu, Shunji
dc.contributor.authorOtero Espinar, Francisco Javier
dc.contributor.authorCouce Pico, María de la Luz
dc.date.accessioned2020-04-07T16:34:15Z
dc.date.available2020-04-07T16:34:15Z
dc.date.issued2019
dc.identifier.citationÁlvarez, J.V.; Bravo, S.B.; García-Vence, M.; De Castro, M.J.; Luzardo, A.; Colón, C.; Tomatsu, S.; Otero-Espinar, F.J.; Couce, M.L. Proteomic Analysis in Morquio A Cells Treated with Immobilized Enzymatic Replacement Therapy on Nanostructured Lipid Systems. Int. J. Mol. Sci. 2019, 20, 4610
dc.identifier.urihttp://hdl.handle.net/10347/21229
dc.description.abstract[ENG]Morquio A syndrome, or mucopolysaccharidosis type IVA (MPS IVA), is a lysosomal storage disease due to mutations in the N-acetylgalactosamine-6-sulfatase (GALNS) gene. Systemic skeletal dysplasia and the related clinical features of MPS IVA are due to disruption of cartilage and its extracellular matrix, leading to an imbalance of growth. Enzyme replacement therapy (ERT) with recombinant human GALNS, alpha elosulfase, provides a systemic treatment. However, this therapy has a limited impact on skeletal dysplasia because the infused enzyme cannot penetrate cartilage and bone. Therefore, an alternative therapeutic approach to reach the cartilage is an unmet challenge. We have developed a new drug delivery system based on a nanostructure lipid carrier with the capacity to immobilize enzymes used for ERT and to target the lysosomes. This study aimed to assess the effect of the encapsulated enzyme in this new delivery system, using in vitro proteomic technology. We found a greater internalization of the enzyme carried by nanoparticles inside the cells and an improvement of cellular protein routes previously impaired by the disease, compared with conventional ERT. This is the first qualitative and quantitative proteomic assay that demonstrates the advantages of a new delivery system to improve the MPS IVA ERT
dc.language.isoeng
dc.publisherMDPI
dc.rights© 2019 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 (http://creativecommons.org/licenses/by/4.0/)
dc.rights.urihttps://creativecommons.org/licenses/by/4.0/
dc.subjectEnzyme replacement therapy
dc.subjectLysosomal disorders
dc.subjectNanoparticles
dc.subjectProteomics
dc.titleProteomic analysis in morquio a cells treated with immobilized enzymatic replacement therapy on nanostructured lipid systems
dc.typeinfo:eu-repo/semantics/article
dc.identifier.DOI10.3390/ijms20184610
dc.relation.publisherversionhttps://www.mdpi.com/1422-0067/20/18/4610
dc.type.versioninfo:eu-repo/semantics/publishedVersion
dc.identifier.e-issn1422-0067
dc.rights.accessrightsinfo:eu-repo/semantics/openAccess
dc.contributor.affiliationUniversidade de Santiago de Compostela. Departamento de Farmacoloxía, Farmacia e Tecnoloxía Farmacéutica
dc.description.peerreviewedSI


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© 2019 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 (http://creativecommons.org/licenses/by/4.0/)
Except where otherwise noted, this item's license is described as  © 2019 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 (http://creativecommons.org/licenses/by/4.0/)





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