Ubiquitous Transgene Expression of the Glucosylceramide-Synthesizing Enzyme Accelerates Glucosylceramide Accumulation and Storage Cells in a Gaucher Disease Mouse Model

Gaucher disease is a lysosomal storage disease caused by defective activity of acid β-glucosidase (GCase), which leads to the accumulation of its major substrates, glucosylceramide (GlcCer) and glucosylsphingosine (GlcSph) in many cells. To modulate cellular substrate concentration in viable mouse m...

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Bibliographic Details
Published in:PLoS One
Main Authors: Barnes, Sonya, Xu, You-Hai, Zhang, Wujuan, Liou, Benjamin, Setchell, Kenneth D. R., Bao, Liming, Grabowski, Gregory A., Sun, Ying
Format: Artigo
Language:Inglês
Published: Public Library of Science 2014
Subjects:
Research Article
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC4281226/
https://ncbi.nlm.nih.gov/pubmed/25551612
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0116023
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