Literature DB >> 28600215

A new type of pharmacological chaperone for GM1-gangliosidosis related human lysosomal β-galactosidase: N-Substituted 5-amino-1-hydroxymethyl-cyclopentanetriols.

Michael Schalli1, Patrick Weber1, Christina Tysoe2, Bettina M Pabst3, Martin Thonhofer1, Eduard Paschke3, Arnold E Stütz4, Marion Tschernutter3, Werner Windischhofer3, Stephen G Withers2.   

Abstract

N-Functionalized amino(hydroxymethyl)cyclopentanetriols are potent inhibitors of β-d-galactosidases and, for the first time, could be shown to act as pharmacological chaperones for GM1-gangliosidosis-associated lysosomal acid β-galactosidase thus representing a new structural type of pharmacological chaperones for this lysosomal storage disease.
Copyright © 2017 Elsevier Ltd. All rights reserved.

Entities:  

Keywords:  Aminocyclopentane; Carbafuranose; G(M1)-Gangliosidosis; Galactosidase inhibitor; Pharmacological chaperone

Mesh：

Substances：

Year:  2017        PMID： 28600215     DOI： 10.1016/j.bmcl.2017.05.086

Source DB:  PubMed          Journal:  Bioorg Med Chem Lett        ISSN： 0960-894X            Impact factor:   2.823


  5 in total

1.  Axonopathy and Reduction of Membrane Resistance: Key Features in a New Murine Model of Human GM1-Gangliosidosis.

Authors:  Deborah Eikelberg; Annika Lehmbecker; Graham Brogden; Witchaya Tongtako; Kerstin Hahn; Andre Habierski; Julia B Hennermann; Hassan Y Naim; Felix Felmy; Wolfgang Baumgärtner; Ingo Gerhauser
Journal:  J Clin Med       Date:  2020-04-02       Impact factor: 4.241

2.  Polyhydroxylated Cyclopentane β-Amino Acids Derived from d-Mannose and d-Galactose: Synthesis and Protocol for Incorporation into Peptides.

Authors:  Fernando Fernández; Alberto G Fernández; Rosalino Balo; Víctor M Sánchez-Pedregal; Miriam Royo; Raquel G Soengas; Ramón J Estévez; Juan C Estévez
Journal:  ACS Omega       Date:  2022-01-04

3.  New α-galactosidase-inhibiting aminohydroxycyclopentanes.

Authors:  Patrick Weber; Roland Fischer; Seyed A Nasseri; Arnold E Stütz; Martin Thonhofer; Stephen G Withers; Andreas Wolfsgruber; Tanja M Wrodnigg
Journal:  RSC Adv       Date:  2021-04-29       Impact factor: 4.036

4.  Potent GH20 N-Acetyl-β-d-hexosaminidase Inhibitors: N-Substituted 3-acetamido-4-amino-5-hydroxymethyl-cyclopentanediols.

Authors:  Patrick Weber; Seyed A Nasseri; Bettina M Pabst; Ana Torvisco; Philipp Müller; Eduard Paschke; Marion Tschernutter; Werner Windischhofer; Stephen G Withers; Tanja M Wrodnigg; Arnold E Stütz
Journal:  Molecules       Date:  2018-03-20       Impact factor: 4.411

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

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
Journal:  Molecules       Date:  2020-09-03       Impact factor: 4.411
  5 in total

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