RT Journal Article
T1 Proteomic analysis in morquio a cells treated with immobilized enzymatic replacement therapy on nanostructured lipid systems
A1 Álvarez González, José Víctor
A1 Bravo López, Susana Belén
A1 García Vence, María
A1 Castro López, María José de
A1 Luzardo Álvarez, Asteria María
A1 Colón Mejeras, Cristóbal
A1 Tomatsu, Shunji
A1 Otero Espinar, Francisco Javier
A1 Couce Pico, María Luz
K1 Enzyme replacement therapy
K1 Lysosomal disorders
K1 Nanoparticles
K1 Proteomics
AB [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
PB MDPI
YR 2019
FD 2019
LK http://hdl.handle.net/10347/21229
UL http://hdl.handle.net/10347/21229
LA eng
NO Álvarez, 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
DS Minerva
RD 27 abr 2026