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N-Butyl-L-Deoxynojirimycin (L-NBDNJ): Synthesis of an allosteric enhancer of α-glucosidase activity for the treatment of Pompe disease

Abstract:
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.
Publication status:
Published
Peer review status:
Peer reviewed
Version:
Accepted manuscript

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Publisher copy:
10.1021/acs.jmedchem.7b00646

Authors


D'Alonzo, D More by this author
De Fenza, M More by this author
More by this author
Institution:
University of Oxford
Division:
Medical Sciences Division
Department:
Pharmacology
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Italian Ministry of Education, Universities and Research More from this funder
Mizutani Foundation for Glycoscience More from this funder
Royal Society More from this funder
Wellcome Trust More from this funder
Publisher:
American Chemical Society Publisher's website
Journal:
Journal of Medicinal Chemistry Journal website
Volume:
60
Issue:
23
Pages:
9462–9469
Publication date:
2017-11-07
Acceptance date:
2017-11-07
DOI:
EISSN:
1520-4804
ISSN:
0022-2623
Pubs id:
pubs:743861
URN:
uri:ab4e4749-166a-4141-8afd-4f3253fe1a44
UUID:
uuid:ab4e4749-166a-4141-8afd-4f3253fe1a44
Local pid:
pubs:743861

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