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Journal article

Beneficial effects of substrate reduction therapy in a mouse model of GM1 gangliosidosis.

Abstract:

GM1 gangliosidosis is an inherited neurodegenerative disorder caused by lysosomal beta-galactosidase deficiency, resulting in the storage of GM1 and GA1, primarily in the central nervous system. This disease typically afflicts infants and young children and there is currently no effective therapy. Substrate reduction therapy (SRT) could be of potential benefit. The imino sugars N-butyldeoxynojirimycin (NB-DNJ, miglustat, Zavesca) and N-butyldeoxygalactonojirimycin (NB-DGJ) used for SRT inhibi...

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Publication status:
Published

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Publisher copy:
10.1016/j.ymgme.2008.02.005

Authors


Elliot-Smith, E More by this author
Lloyd-Evans, E More by this author
van der Spoel, AC More by this author
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Journal:
Molecular genetics and metabolism
Volume:
94
Issue:
2
Pages:
204-211
Publication date:
2008-06-05
DOI:
EISSN:
1096-7206
ISSN:
1096-7192
URN:
uuid:205652a2-14ab-45b6-8a66-e8005825e857
Source identifiers:
101081
Local pid:
pubs:101081

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