Substrate reduction therapy in mouse models of the glycosphingolipidoses.

Substrate reduction therapy uses small molecules to slow the rate of glycolipid biosynthesis. One of these drugs, N-butyldeoxynojirimycin (NB-DNJ), shows efficacy in mouse models of Tay-Sachs, Sandhoff and Fabry diseases. This offers the prospect that NB-DNJ may be of therapeutic benefit, at least i...

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Detalhes bibliográficos
Main Authors: Platt, Frances M, Jeyakumar, Mylvaganam, Andersson, Ulrika, Heare, Tanya, Dwek, Raymond A, Butters, Terry D
Formato: Artigo
Idioma:Inglês
Publicado em: 2003
Assuntos:
Research Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC1693185/
https://ncbi.nlm.nih.gov/pubmed/12803928
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1098/rstb.2003.1279
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