N-Butyl-L-Deoxynojirimycin (L-NBDNJ): Synthesis of an allosteric enhancer of α-glucosidase activity for the treatment of Pompe disease
The highly stereocontrolled de novo synthesis of L-NBDNJ (the unnatural enantiomer of the iminosugar drug Miglustat) and a preliminary evaluation of its chaperoning potential are herein reported. L-NBDNJ is able to enhance lysosomal α-glucosidase levels in Pompe disease fibroblasts, either when admi...
| Main Authors: | , , , , , , , , , , , |
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| Format: | Journal article |
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American Chemical Society
2017
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| _version_ | 1826290406222987264 |
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| author | D'Alonzo, D De Fenza, M Porto, C Iacono, R Huebecker, M Cobucci-Ponzano, B Priestman, D Platt, F Parenti, G Moracci, M Palumbo, G Guaragna, A |
| author_facet | D'Alonzo, D De Fenza, M Porto, C Iacono, R Huebecker, M Cobucci-Ponzano, B Priestman, D Platt, F Parenti, G Moracci, M Palumbo, G Guaragna, A |
| author_sort | D'Alonzo, D |
| collection | OXFORD |
| description | The highly stereocontrolled de novo synthesis of L-NBDNJ (the unnatural enantiomer of the iminosugar drug Miglustat) and a preliminary evaluation of its chaperoning potential are herein reported. L-NBDNJ is able to enhance lysosomal α-glucosidase levels in Pompe disease fibroblasts, either when administered singularly or when co-incubated with the recombinant human α-glucosidase. In addition, differently from its D-enantiomer, L-NBDNJ does not act as a glycosidase inhibitor. |
| first_indexed | 2024-03-07T02:43:42Z |
| format | Journal article |
| id | oxford-uuid:ab4e4749-166a-4141-8afd-4f3253fe1a44 |
| institution | University of Oxford |
| last_indexed | 2024-03-07T02:43:42Z |
| publishDate | 2017 |
| publisher | American Chemical Society |
| record_format | dspace |
| spelling | oxford-uuid:ab4e4749-166a-4141-8afd-4f3253fe1a442022-03-27T03:21:09ZN-Butyl-L-Deoxynojirimycin (L-NBDNJ): Synthesis of an allosteric enhancer of α-glucosidase activity for the treatment of Pompe diseaseJournal articlehttp://purl.org/coar/resource_type/c_dcae04bcuuid:ab4e4749-166a-4141-8afd-4f3253fe1a44Symplectic Elements at OxfordAmerican Chemical Society2017D'Alonzo, DDe Fenza, MPorto, CIacono, RHuebecker, MCobucci-Ponzano, BPriestman, DPlatt, FParenti, GMoracci, MPalumbo, GGuaragna, AThe highly stereocontrolled de novo synthesis of L-NBDNJ (the unnatural enantiomer of the iminosugar drug Miglustat) and a preliminary evaluation of its chaperoning potential are herein reported. L-NBDNJ is able to enhance lysosomal α-glucosidase levels in Pompe disease fibroblasts, either when administered singularly or when co-incubated with the recombinant human α-glucosidase. In addition, differently from its D-enantiomer, L-NBDNJ does not act as a glycosidase inhibitor. |
| spellingShingle | D'Alonzo, D De Fenza, M Porto, C Iacono, R Huebecker, M Cobucci-Ponzano, B Priestman, D Platt, F Parenti, G Moracci, M Palumbo, G Guaragna, A N-Butyl-L-Deoxynojirimycin (L-NBDNJ): Synthesis of an allosteric enhancer of α-glucosidase activity for the treatment of Pompe disease |
| title | N-Butyl-L-Deoxynojirimycin (L-NBDNJ): Synthesis of an allosteric enhancer of α-glucosidase activity for the treatment of Pompe disease |
| title_full | N-Butyl-L-Deoxynojirimycin (L-NBDNJ): Synthesis of an allosteric enhancer of α-glucosidase activity for the treatment of Pompe disease |
| title_fullStr | N-Butyl-L-Deoxynojirimycin (L-NBDNJ): Synthesis of an allosteric enhancer of α-glucosidase activity for the treatment of Pompe disease |
| title_full_unstemmed | N-Butyl-L-Deoxynojirimycin (L-NBDNJ): Synthesis of an allosteric enhancer of α-glucosidase activity for the treatment of Pompe disease |
| title_short | N-Butyl-L-Deoxynojirimycin (L-NBDNJ): Synthesis of an allosteric enhancer of α-glucosidase activity for the treatment of Pompe disease |
| title_sort | n butyl l deoxynojirimycin l nbdnj synthesis of an allosteric enhancer of α glucosidase activity for the treatment of pompe disease |
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