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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Bibliografische gegevens
Gepubliceerd in:EBioMedicine
Hoofdauteurs: Sun, Ying, Liou, Benjamin, Chu, Zhengtao, Fannin, Venette, Blackwood, Rachel, Peng, Yanyan, Grabowski, Gregory A., Davis, Harold W., Qi, Xiaoyang
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: Elsevier 2020
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Research paper
Online toegang: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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