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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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| Autors principals: | , , , , , |
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| Format: | Artigo |
| Idioma: | Inglês |
| Publicat: |
2003
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| Matèries: | |
| Accés en línia: | 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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