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• W2023238175 abstract "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." @default.
• W2023238175 created "2016-06-24" @default.
• W2023238175 creator A5062018516 @default.
• W2023238175 creator A5076658123 @default.
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• W2023238175 date "2003-02-01" @default.
• W2023238175 modified "2023-10-17" @default.
• W2023238175 title "Therapeutic Applications of Imino Sugars in Lysosomal Storage Disorders" @default.
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• W2023238175 doi "https://doi.org/10.2174/1568026033452483" @default.
• W2023238175 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/12570866" @default.
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