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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 1 Gaucher disease and has shown to be an effective therapy for this disorder.

Type

Journal article

Journal

Curr Top Med Chem

Publication Date

2003

Volume

3

Pages

561 - 574

Keywords

Animals, Glycolipids, Humans, Lysosomal Storage Diseases, Monosaccharides, Structure-Activity Relationship