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Enzyme-Loaded Gel Core Nanostructured Lipid Carriers to Improve Treatment of Lysosomal Storage Diseases: Formulation and In Vitro Cellular Studies of Elosulfase Alfa-Loaded Systems

Mucopolysaccharidosis IVA (Morquio A) is a rare inherited metabolic disease caused by deficiency of the lysosomal enzyme N-acetylgalatosamine-6-sulfate-sulfatase (GALNS). Until now, treatments employed included hematopoietic stem cell transplantation and enzyme replacement therapy (ERT); the latter...

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Detalles Bibliográficos
Autores principales: Álvarez, J. Víctor, Herrero Filgueira, Carolina, González, Alexandre de la Fuente, Colón Mejeras, Cristóbal, Beiras Iglesias, Andrés, Tomatsu, Shunji, Blanco Méndez, José, Luzardo Álvarez, Asteria, Couce, María Luz, Otero Espinar, Francisco J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2019
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6835858/
https://www.ncbi.nlm.nih.gov/pubmed/31614479
http://dx.doi.org/10.3390/pharmaceutics11100522