Substrate reduction therapy in mouse models of the glycosphingolipidoses
| Autor: | Frances M. Platt, Tanya Heare, Raymond A. Dwek, Mylvaganam Jeyakumar, Terry D. Butters, Ulrika Andersson |
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| Jazyk: | angličtina |
| Rok vydání: | 2016 |
| Předmět: |
Drug
1-Deoxynojirimycin media_common.quotation_subject Biology Pharmacology General Biochemistry Genetics and Molecular Biology Glycolipid biosynthesis Sphingolipidoses Substrate Specificity chemistry.chemical_compound Mice medicine Animals Substrate reduction therapy Dosing Enzyme Inhibitors media_common medicine.disease Disease Models Animal chemistry Substrate specificity Infantile onset General Agricultural and Biological Sciences Research Article |
| Popis: | 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 in the juvenile and adult onset variants of these disorders. The infantile onset variants will require an additional enzyme–augmenting modality if the pathology is to be significantly improved. A second drug, N –butyldeoxyglactonojirimycin, looks very promising for treating storage diseases with neurological involvement as high systemic dosing is achievable without any side–effects. |
| Databáze: | OpenAIRE |
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