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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Udgivet i:EBioMedicine
Main Authors: Sun, Ying, Liou, Benjamin, Chu, Zhengtao, Fannin, Venette, Blackwood, Rachel, Peng, Yanyan, Grabowski, Gregory A., Davis, Harold W., Qi, Xiaoyang
Format: Artigo
Sprog:Inglês
Udgivet: Elsevier 2020
Fag:
Research paper
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7251241/
https://ncbi.nlm.nih.gov/pubmed/32279952
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ebiom.2020.102735
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