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Systemic enzyme delivery by blood-brain barrier-penetrating SapC-DOPS nanovesicles for treatment of neuronopathic Gaucher disease

BACKGROUND: Enzyme replacement therapy (ERT) can positively affect the visceral manifestations of lysosomal storage diseases (LSDs). However, the exclusion of the intravenous ERT agents from the central nervous system (CNS) prevents direct therapeutic effects. METHODS: Using a neuronopathic Gaucher...

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Detalles Bibliográficos
Autores principales:	Sun, Ying, Liou, Benjamin, Chu, Zhengtao, Fannin, Venette, Blackwood, Rachel, Peng, Yanyan, Grabowski, Gregory A., Davis, Harold W., Qi, Xiaoyang
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Elsevier 2020
Materias:	Research paper
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7251241/ https://www.ncbi.nlm.nih.gov/pubmed/32279952 http://dx.doi.org/10.1016/j.ebiom.2020.102735

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