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From the same journal

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

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  • 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


Publication details

DateAccepted/In press - 25 Aug 2020
DatePublished (current) - 3 Sep 2020
Issue number17
Number of pages25
Original languageEnglish


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 of lipophilic analogs of the potent β-d-galactosidase inhibitor 4-epi-isofagomine. Structure-activity relationships were investigated by X-ray crystallography as well as by alterations in the cyclopentane moiety such as deoxygenation and replacement by fluorine of a “strategic” hydroxyl group. New compounds have revealed highly promising activities with a range of β-galactosidase-compromised human cell lines and may serve as leads towards new pharmacological chaperones for GM1-gangliosidosis and Morquio B disease.

Bibliographical note

© 2020 by the authors.

    Research areas

  • 4-epi-isofagomine, Aminocyclopentane, Carbasugar, G-gangliosidosis, Galactosidase inhibitor, Iminoalditol, Pharmacological chaperone


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