The DOI matches its Crossref record; 10 of the 10 references it cites were found in a registry or an index.
Prevention of Lysosomal Storage in Tay-Sachs Mice Treated with N -Butyldeoxynojirimycin
Abstract
The glycosphingolipid (GSL) lysosomal storage diseases result from the inheritance of defects in the genes encoding the enzymes required for catabolism of GSLs within lysosomes. A strategy for the treatment of these diseases, based on an inhibitor of GSL biosynthesis N -butyldeoxynojirimycin, was evaluated in a mouse model of Tay-Sachs disease. When Tay-Sachs mice were treated with N -butyldeoxynojirimycin, the accumulation of G M2 in the brain was prevented, with the number of storage neurons and the quantity of ganglioside stored per cell markedly reduced. Thus, limiting the biosynthesis of the substrate (G M2 ) for the defective enzyme (β-hexosaminidase A) prevents GSL accumulation and the neuropathology associated with its lysosomal storage.
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Self-citationN-butyldeoxynojirimycin is a novel inhibitor of glycolipid biosynthesis.
Platt, F. M., Neises, G. R., Dwek, R. A., & Butters, T. D. (1994) · Journal of Biological Chemistry · doi:10.1016/s0021-9258(17)37202-2 · Cited by 455
Self-citationYamanaka, S., Johnson, M. D., Grinberg, A., Westphal, H., Crawley, J. N., Taniike, M., Suzuki, K., & Proia, R. L. (1994) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.91.21.9975 · Cited by 209
Self-citationGaucher Disease: New Molecular Approaches to Diagnosis and Treatment
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Neuropathology of mice with targeted disruption of Hexa gene, a model of Tay-Sachs disease
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Self-citationInhibitors of Glycosphingolipid Biosynthesis.
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Asano, N., Nash, R. J., Molyneux, R. J., & Fleet, G. W. J. (2000) · Tetrahedron: Asymmetry · doi:10.1016/s0957-4166(00)00113-0 · Cited by 1,015
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Self-citationPolyhydroxylated alkaloids—Natural occurrence and therapeutic applications
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Cu(I)-Catalyzed Click Chemistry in Glycoscience and Their Diverse Applications
Agrahari, A. K., Bose, P., Jaiswal, M. K., Rajkhowa, S., Singh, A. S., Hotha, S., Mishra, N., & Tiwari, V. K. (2021) · Chemical Reviews · doi:10.1021/acs.chemrev.0c00920 · Cited by 445
Targeting glycosylation as a therapeutic approach
Dwek, R. A., Butters, T. D., Platt, F. M., & Zitzmann, N. (2002) · Nature Reviews Drug Discovery · doi:10.1038/nrd708 · Cited by 420
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Metadata
- Authors
- Platt, F. M., Neises, G. R., Reinkensmeier, G., Townsend, M. J., Perry, V. H., Proia, R. L., Winchester, B., Dwek, R. A., & Butters, T. D.
- Venue
- Science
- Published
- 1997-04-18
- Volume
- 276
- Issue
- 5311
- Pages
- 428-431
- ISSN
- 0036-8075 (ISSN-L)
- 1095-9203
- OpenAlex
- W2032826794
- Cited by
- 340
- References
- 10
- FWCI
- 13.78
- Type
- journal article
- Language
- en
- Open access
- closed
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- CoChat. (2026). Citation check: Prevention of Lysosomal Storage in Tay-Sachs Mice Treated with N -Butyldeoxynojirimycin. https://app.cochat.ai/papers/10.1126/science.276.5311.428
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- Platt, F. M., Neises, G. R., Reinkensmeier, G., Townsend, M. J., Perry, V. H., Proia, R. L., Winchester, B., Dwek, R. A., & Butters, T. D. (1997). Prevention of Lysosomal Storage in Tay-Sachs Mice Treated with N -Butyldeoxynojirimycin. Science. https://doi.org/10.1126/science.276.5311.428
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Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
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