D'Alonzo D., De Fenza M., Porto C., Iacono R., Huebecker M., Cobucci-Ponzano B., Priestman DA., Platt F., Parenti G., Moracci M., Palumbo G., Guaragna A.

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.

N-Butyl-l-deoxynojirimycin (l-NBDNJ): Synthesis of an Allosteric Enhancer of α-Glucosidase Activity for the Treatment of Pompe Disease.

D'Alonzo D., De Fenza M., Porto C., Iacono R., Huebecker M., Cobucci-Ponzano B., Priestman DA., Platt F., Parenti G., Moracci M., Palumbo G., Guaragna A.

DOI

Type

Journal

Publication Date

Volume

Pages

Keywords