Improved outcome of N-butyldeoxygalactonojirimycin-mediated substrate reduction therapy in a mouse model of Sandhoff disease

Improved outcome of N-butyldeoxygalactonojirimycin-mediated substrate reduction therapy in a mouse model of Sandhoff disease

Sandhoff disease is a severe neurodegenerative glycosphingolipid (GSL) lysosomal storage disorder, currently without treatment options. One therapeutic approach under investigation is substrate reduction therapy (SRT). By partially inhibiting GSL biosynthesis, the impaired rate of GSL catabolism is...

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Bibliographic Details
Main Authors: Ulrika Andersson, David Smith, Mylvaganam Jeyakumar, Terry D Butters, Mario Cortina Borja, Raymond A Dwek, Frances M Platt
Format: Article
Language:English
Published: Elsevier 2004-08-01
Series:Neurobiology of Disease
Subjects:
Sandhoff disease
Gangliosidosis
Substrate reduction therapy
Imino sugar
Online Access:http://www.sciencedirect.com/science/article/pii/S0969996104001056

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http://www.sciencedirect.com/science/article/pii/S0969996104001056

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