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Design,Synthesis, and Biological Evaluation of Enantiomeric β‐N‐Acetylhexosaminidase Inhibitors LABNAc and DABNAc as Potential Agents against Tay‐Sachs and Sandhoff Disease |
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| Authors: | J. S. Shane Rountree Dr. Terry D. Butters Dr. Mark R. Wormald Dr. Stephanie D. Boomkamp Raymond A. Dwek Prof. Naoki Asano Prof. Kyoko Ikeda Dr. Emma L. Evinson Dr. Robert J. Nash Dr. George W. J. Fleet Prof. |
| Affiliation: | 1. Chemistry Research Laboratory, Department of Chemistry, University of Oxford, Mansfield Road, Oxford, OX1?3TA (UK), Fax (G.W.J.F.): (+44)?1865‐277386;2. Oxford Glycobiology Institute, Department of Biochemistry, University of Oxford, South Parks Road, Oxford, OX1?3QU (UK), Fax (T.D.B.): (+44)?1865‐275216;3. Department of Biochemistry, Faculty of Pharmaceutical Sciences, Hokuriku University, Kanazawa, Ishikawa 9201181 (Japan);4. Institute of Grassland and Environmental Research, Plas Gogerddan, Aberystwyth SY23?3EB, Dyfed, Wales (UK) |
| Abstract: | Combating glycolipid storage disorders : LABNAc was prepared in an efficient 11‐step procedure from D ‐lyxonolactone. The enantiomer DABNAc was also prepared from L ‐lyxonolactone. Preliminary cellular studies indicate that these compounds may find utility as chemical chaperones for the treatment of Tay‐Sachs and Sandhoff diseases.  |
| Keywords: | azasugars chaperone proteins GM2 gangliosidoses inhibitors sugar lactones |