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SHR Neuro Krebs Kardio Lipid Stoffw Microb

Weber, P; Thonhofer, M; Averill, S; Davies, GJ; Santana, AG; Müller, P; Nasseri, SA; Offen, WA; Pabst, BM; Paschke, E; Schalli, M; Torvisco, A; Tschernutter, M; Tysoe, C; Windischhofer, W; Withers, SG; Wolfsgruber, A; Wrodnigg, TM; Stütz, AE.
Mechanistic Insights into the Chaperoning of Human Lysosomal-Galactosidase Activity: Highly Functionalized Aminocyclopentanes and C-5a-Substituted Derivatives of 4-epi-Isofagomine.
Molecules. 2020; 25(17): Doi: 10.3390/molecules25174025 [OPEN ACCESS]
Web of Science PubMed PUBMED Central FullText FullText_MUG


Co-Autor*innen der Med Uni Graz
Pabst Bettina
Paschke Eduard
Schalli Michael
Tschernutter Marion
Windischhofer Werner

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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.

Find related publications in this database (Keywords)
galactosidase inhibitor
pharmacological chaperone
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