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

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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Bibliographic Details
Main Authors: Ying Sun, Benjamin Liou, Zhengtao Chu, Venette Fannin, Rachel Blackwood, Yanyan Peng, Gregory A. Grabowski, Harold W. Davis, Xiaoyang Qi
Format: Article
Language:English
Published: Elsevier 2020-05-01
Series:EBioMedicine
Subjects:
Neurodegenerative disease
Central nervous system
SapC-DOPS nanovesicles
Enzyme replacement therapy
Neuronopathic Gaucher disease
Acid-β-glucosidase
Online Access:http://www.sciencedirect.com/science/article/pii/S2352396420301109

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http://www.sciencedirect.com/science/article/pii/S2352396420301109

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