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Umbilical mesenchymal stem cell-derived extracellular vesicles as enzyme delivery vehicle to treat Morquio A fibroblasts

BACKGROUND: Mucopolysaccharidosis IVA (Morquio A syndrome) is a lysosomal storage disease caused by the deficiency of enzyme N-acetylgalactosamine-6-sulfate sulfatase (GALNS), which results in the accumulation of the glycosaminoglycans (GAGs), keratan sulfate, and chondroitin-6-sulfate in the lysoso...

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Detalles Bibliográficos
Autores principales: Flanagan, Michael, Pathak, Isha, Gan, Qi, Winter, Linda, Emnet, Ryan, Akel, Salem, Montaño, Adriana M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8101245/
https://www.ncbi.nlm.nih.gov/pubmed/33957983
http://dx.doi.org/10.1186/s13287-021-02355-0