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Substrate reduction therapy for glycosphingolipid storage disorders.

Abstract:

Substrate reduction therapy is a novel approach to treating glycosphingolipid (GSL) lysosomal storage disorders. These diseases are caused by mutations in the genes coding for enzymes involved in GSL catabolism and are characterised by the accumulation of GSL substrates within the lysosomes of cells. The aim of substrate reduction therapy is to inhibit the rate of synthesis of GSLs to levels where the residual activity of the mutant catabolic enzyme is sufficient to prevent pathological stora...

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Publication status:
Published

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Publisher copy:
10.1517/13543784.10.3.455

Authors


Lachmann, RH More by this author
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Institution:
University of Oxford
Department:
Oxford, MSD, Pharmacology
Journal:
Expert opinion on investigational drugs
Volume:
10
Issue:
3
Pages:
455-466
Publication date:
2001-03-05
DOI:
EISSN:
1744-7658
ISSN:
1354-3784
URN:
uuid:499dc838-720f-47c0-ae7c-e7919a9e8124
Source identifiers:
185331
Local pid:
pubs:185331

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