Cyclodextrin-Based Iminosugar Click Clusters: The First Examples of Multivalent Pharmacological Chaperones for the Treatment of Lysosomal Storage Disorders

Seven‐arm chaperone: A modest μM inhibitor of glucosylceramide β‐glucosidase (GCase) has been transformed into a potent low‐nM inhibitor by multivalency. This iminosugar inhibitor acts as a pharmacological chaperone and increases residual GCase activity in fibroblasts from Gaucher patients. These re...

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Veröffentlicht in:	Chembiochem : a European journal of chemical biology 2012-03, Vol.13 (5), p.661-664
Hauptverfasser:	Decroocq, Camille, Rodríguez-Lucena, David, Ikeda, Kyoko, Asano, Naoki, Compain, Philippe
Format:	Artikel
Sprache:	eng
Schlagworte:	chaperones Click Chemistry cyclodextrins Cyclodextrins - chemistry Cyclodextrins - pharmacology Cyclodextrins - therapeutic use Gaucher's disease Humans Imino Sugars - chemistry Imino Sugars - pharmacology Imino Sugars - therapeutic use iminosugars Lysosomal Storage Diseases - drug therapy Molecular Chaperones - chemistry Molecular Chaperones - pharmacology Molecular Chaperones - therapeutic use Molecular Structure multivalency
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Zusammenfassung:	Seven‐arm chaperone: A modest μM inhibitor of glucosylceramide β‐glucosidase (GCase) has been transformed into a potent low‐nM inhibitor by multivalency. This iminosugar inhibitor acts as a pharmacological chaperone and increases residual GCase activity in fibroblasts from Gaucher patients. These results open the way to a new class of chaperones for the treatment of lysosomal diseases.
ISSN:	1439-4227 1439-7633
DOI:	10.1002/cbic.201200005