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Chemical chaperone therapy for brain pathology in G(M1)-gangliosidosis
We synthesized a galactose derivative, N-octyl-4-epi-β-valienamine (NOEV), for a molecular therapy (chemical chaperone therapy) of a human neurogenetic disease, β-galactosidosis (G(M1)-gangliosidosis and Morquio B disease). It is a potent inhibitor of lysosomal β-galactosidase in vitro. Addition of...
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| Main Authors: | , , , , , , , , , , , , , , , , , , , |
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| Format: | Artigo |
| Sprog: | Inglês |
| Udgivet: |
National Academy of Sciences
2003
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| Fag: | |
| Online adgang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC307667/ https://ncbi.nlm.nih.gov/pubmed/14676316 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.2536657100 |
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