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N-butyldeoxygalactonojirimycin reduces brain ganglioside and GM2 content in neonatal Sandhoff disease mice.

Abstract:

Sandhoff disease involves the CNS accumulation of ganglioside GM2 and asialo-GM2 (GA2) due to inherited defects in the beta-subunit gene of beta-hexosaminidase A and B (Hexb gene). Accumulation of these glycosphingolipids (GSLs) produces progressive neurodegeneration, ultimately leading to death. Substrate reduction therapy (SRT) aims to decrease the rate of glycosphingolipid (GSL) biosynthesis to compensate for the impaired rate of catabolism. The imino sugar, N-butyldeoxygalactonojirimycin ...

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Institution:
University of Oxford
Division:
MSD
Department:
Pharmacology
Role:
Author
Journal:
Neurochemistry international More from this journal
Volume:
52
Issue:
6
Pages:
1125-1133
Publication date:
2008-05-01
DOI:
EISSN:
1872-9754
ISSN:
0197-0186
Language:
English
Keywords:
Pubs id:
pubs:185359
UUID:
uuid:24b0d2ac-f74e-4546-87ba-7954dceda9f1
Local pid:
pubs:185359
Source identifiers:
185359
Deposit date:
2012-12-19

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