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Journal article

Substrate reduction therapy in mouse models of the glycosphingolipidoses.

Abstract:

Substrate reduction therapy uses small molecules to slow the rate of glycolipid biosynthesis. One of these drugs, N-butyldeoxynojirimycin (NB-DNJ), shows efficacy in mouse models of Tay-Sachs, Sandhoff and Fabry diseases. This offers the prospect that NB-DNJ may be of therapeutic benefit, at least in the juvenile and adult onset variants of these disorders. The infantile onset variants will require an additional enzyme-augmenting modality if the pathology is to be significantly improved. A se...

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Publication status:
Published

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Publisher copy:
10.1098/rstb.2003.1279

Authors


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Institution:
University of Oxford
Department:
Oxford, MSD, Pharmacology
Jeyakumar, M More by this author
Andersson, U More by this author
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Journal:
Philosophical transactions of the Royal Society of London. Series B, Biological sciences
Volume:
358
Issue:
1433
Pages:
947-954
Publication date:
2003-05-05
DOI:
EISSN:
1471-2970
ISSN:
0962-8436
URN:
uuid:3539421f-78bb-40c4-a382-aa86b04104a2
Source identifiers:
100984
Local pid:
pubs:100984

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