Journal
CURRENT TOPICS IN MEDICINAL CHEMISTRY
Volume 3, Issue 5, Pages 561-574Publisher
BENTHAM SCIENCE PUBL LTD
DOI: 10.2174/1568026033452483
Keywords
lysosomal; glycosidase; glucosyltransferase; glycolipid; inhibitor; substrate reduction therapy
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The N-alkylated imino sugars have inhibitory activity against the first enzyme in the pathway for glucosylating sphingolipid in eukaryotic cells, ceramide-specific glucosyltransferase. A therapeutic approach termed 'substrate deprivation' or,substrate reduction therapy' (SRT) aims to reduce biosynthetic capability in the cell to match the reduced lysosomal catalytic activity seen in lysosomal storage disorders. The use of N-alkylated imino sugars to establish this therapeutic strategy is described in cell culture and gene knockout mouse disease models. One imino sugar, N-butyl-DNJ (NB-DNJ) has been in clinical trials for type I Gaucher disease and has shown to be an effective therapy for this disorder.
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