Medizinische Universität Graz Austria/Österreich - Forschungsportal - Medical University of Graz
Gewählte Publikation:
SHR Neuro Krebs Kardio Lipid Stoffw Microb
Schalli, M; Weber, P; Tysoe, C; Pabst, BM; Thonhofer, M; Paschke, E; Stütz, AE; Tschernutter, M; Windischhofer, W; Withers, SG.
A new type of pharmacological chaperone for GM1-gangliosidosis related human lysosomal β-galactosidase: N-Substituted 5-amino-1-hydroxymethyl-cyclopentanetriols.
Bioorg Med Chem Lett. 2017; 27(15):3431-3435
Doi: 10.1016/j.bmcl.2017.05.086
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:
- 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.
- Find related publications in this database (using NLM MeSH Indexing)
- Amination -
- Animals -
- Cattle -
- Cyclopentanes - chemistry
- Cyclopentanes - pharmacology
- Enzyme Inhibitors - chemistry
- Enzyme Inhibitors - pharmacology
- Gangliosidosis, GM1 - drug therapy
- Gangliosidosis, GM1 - enzymology
- Humans -
- Lysosomes - drug effects
- Lysosomes - enzymology
- Methylation -
- beta-Galactosidase - antagonists & inhibitors
- beta-Galactosidase - metabolism
- Find related publications in this database (Keywords)
- Aminocyclopentane
- Carbafuranose
- Galactosidase inhibitor
- Pharmacological chaperone
- G(M1)-Gangliosidosis