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

Abstract

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. Detailed facts of importance to specialist readers are published as ”Supporting Information”. Such documents are peer-reviewed, but not copy-edited or typeset. They are made available as submitted by the authors. Please note: The publisher is not responsible for the content or functionality of any supporting information supplied by the authors. Any queries (other than missing content) should be directed to the corresponding author for the article.