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Brain-targeted enzyme-loaded nanoparticles: A breach through the blood-brain barrier for enzyme replacement therapy in Krabbe disease

Lysosomal storage disorders (LSDs) result from an enzyme deficiency within lysosomes. The systemic administration of the missing enzyme, however, is not effective in the case of LSDs with central nervous system (CNS)-involvement. Here, an enzyme delivery system based on the encapsulation of cross-li...

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Detalles Bibliográficos
Autores principales: Del Grosso, Ambra, Galliani, Marianna, Angella, Lucia, Santi, Melissa, Tonazzini, Ilaria, Parlanti, Gabriele, Signore, Giovanni, Cecchini, Marco
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Association for the Advancement of Science 2019
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6867879/
https://www.ncbi.nlm.nih.gov/pubmed/31799395
http://dx.doi.org/10.1126/sciadv.aax7462