Mechanistic Insights into the Chaperoning of Human Lysosomal-Galactosidase Activity: Highly Functionalized Aminocyclopentanes and <i>C</i>-5a-Substituted Derivatives of 4-<i>epi</i>-Isofagomine

Mechanistic Insights into the Chaperoning of Human Lysosomal-Galactosidase Activity: Highly Functionalized Aminocyclopentanes and <i>C</i>-5a-Substituted Derivatives of 4-<i>epi</i>-Isofagomine

Glycosidase inhibitors have shown great potential as pharmacological chaperones for lysosomal storage diseases. In light of this, a series of new cyclopentanoid β-galactosidase inhibitors were prepared and their inhibitory and pharmacological chaperoning activities determined and compared with those...

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Bibliographic Details
Main Authors: Patrick Weber, Martin Thonhofer, Summer Averill, Gideon J. Davies, Andres Gonzalez Santana, Philipp Müller, Seyed A. Nasseri, Wendy A. Offen, Bettina M. Pabst, Eduard Paschke, Michael Schalli, Ana Torvisco, Marion Tschernutter, Christina Tysoe, Werner Windischhofer, Stephen G. Withers, Andreas Wolfsgruber, Tanja M. Wrodnigg, Arnold E. Stütz
Format: Article
Language:English
Published: MDPI AG 2020-09-01
Series:Molecules
Subjects:
iminoalditol
4-<i>epi</i>-isofagomine
carbasugar
aminocyclopentane
galactosidase inhibitor
pharmacological chaperone
Online Access:https://www.mdpi.com/1420-3049/25/17/4025

Internet

https://www.mdpi.com/1420-3049/25/17/4025

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