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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...
Autores principales: | Del Grosso, Ambra, Galliani, Marianna, Angella, Lucia, Santi, Melissa, Tonazzini, Ilaria, Parlanti, Gabriele, Signore, Giovanni, Cecchini, Marco |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Association for the Advancement of Science
2019
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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 |
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