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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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Detalhes bibliográficos
Publicado no:Sci Adv
Main Authors: Del Grosso, Ambra, Galliani, Marianna, Angella, Lucia, Santi, Melissa, Tonazzini, Ilaria, Parlanti, Gabriele, Signore, Giovanni, Cecchini, Marco
Formato: Artigo
Idioma:Inglês
Publicado em: American Association for the Advancement of Science 2019
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6867879/
https://ncbi.nlm.nih.gov/pubmed/31799395
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/sciadv.aax7462
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