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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 coincubated with the recombinant human α-glucosidase. In addition, differently from its d-enantiomer, l-NBDNJ does not act as a glycosidase inhibitor.

Original publication

DOI

10.1021/acs.jmedchem.7b00646

Type

Journal article

Journal

Journal of Medicinal Chemistry

Publication Date

12/2017

Volume

60

Pages

9462 - 9469

Addresses

Department of Chemical Sciences, Università degli Studi di Napoli Federico II , via Cintia, 80126 Napoli, Italy.

Keywords

Cell Line, Lysosomes, Fibroblasts, Humans, Glycogen Storage Disease Type II, 1-Deoxynojirimycin, alpha-Glucosidases, Enzyme Inhibitors, Allosteric Regulation, Enzyme Activation, Stereoisomerism, Models, Molecular