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Gewählte Publikation:
SHR Neuro Krebs Kardio Lipid Stoffw Microb
Schalli, M; Tysoe, C; Fischer, R; Pabst, BM; Thonhofer, M; Paschke, E; Rappitsch, T; Stütz, AE; Tschernutter, M; Windischhofer, W; Withers, SG.
N-Substituted 5-amino-1-hydroxymethyl-cyclopentanetriols: A new family of activity promotors for a GM1-gangliosidosis related human lysosomal β-galactosidase mutant.
Carbohydr Res. 2017; 443-444(15):15-22
Doi: 10.1016/j.carres.2017.03.009
Web of Science
PubMed
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- Führende Autor*innen der Med Uni Graz
- Schalli Michael
- Co-Autor*innen der Med Uni Graz
- Pabst Bettina
- Paschke Eduard
- Tschernutter Marion
- Windischhofer Werner
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- Abstract:
- From 1,2;3,4-di-O-isopropylidene-α-D-galactopyranose, a series of highly functionalized (hydroxymethyl)cyclopentanes was easily available. In line with reports by Reymond and Jäger on similar structures, these amine containing basic carbasugars 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 mutant R201C, thus representing a new structural type of pharmacological chaperones for this lysosomal storage disease. Copyright © 2017 Elsevier Ltd. All rights reserved.
- Find related publications in this database (using NLM MeSH Indexing)
- Cyclopentanes - chemical synthesis
- Cyclopentanes - chemistry
- Cyclopentanes - pharmacology
- Enzyme Inhibitors - chemical synthesis
- Enzyme Inhibitors - chemistry
- Enzyme Inhibitors - pharmacology
- Gangliosidosis, GM1 - enzymology
- Gangliosidosis, GM1 - genetics
- Humans -
- Models, Molecular -
- Molecular Conformation -
- Mutation -
- beta-Galactosidase - antagonists & inhibitors
- beta-Galactosidase - genetics
- Find related publications in this database (Keywords)
- Aminocyclopentane
- Carbafuranose
- Galactosidase inhibitor
- Pharmacological chaperone
- G(M1)-Gangliosidosis