Therapeutic Applications of Imino Sugars in Lysosomal Storage Disorders

Title: Therapeutic Applications of Imino Sugars in Lysosomal Storage Disorders

Volume: 3 Issue: 5

Author(s): Terry D. Butters, Raymond A. Dwek and Frances M. Platt

Affiliation:

Keywords: lysosomal, glycosidase, glucosyltransferase, glycolipid, inhibitor, substrate reduction therapy

Abstract: The N-alkylated imino sugars have inhibitory activity against the first enzyme in the pathway for glucosylating sphingolipid in eukaryotic cells, ceramidespecific 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.

Cite this article as:

Butters D. Terry, Dwek A. Raymond and Platt M. Frances, Therapeutic Applications of Imino Sugars in Lysosomal Storage Disorders, Current Topics in Medicinal Chemistry 2003; 3 (5) . https://dx.doi.org/10.2174/1568026033452483