Viral delivery of a microRNA to Gba to the mouse central nervous system models neuronopathic Gaucher disease
Pathological mutations in GBA, encoding lysosomal glucocerebrosidase (GCase), cause Gaucher disease (GD). GD is a multi-system disease with great phenotypic variation between individuals. It has been classified into type 1 with primarily peripheral involvement and types 2 and 3 with varying degrees...
Main Authors: | Kasey L. Jackson, Catherine Viel, Jennifer Clarke, Jie Bu, Monyrath Chan, Bing Wang, Lamya S. Shihabuddin, S. Pablo Sardi |
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Format: | Article |
Language: | English |
Published: |
Elsevier
2019-10-01
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Series: | Neurobiology of Disease |
Subjects: | |
Online Access: | http://www.sciencedirect.com/science/article/pii/S096999611930172X |
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