Issue 1, 2012

Non-iminosugar glucocerebrosidase small molecule chaperones

Abstract

Small molecule chaperones are a promising therapeutic approach for the Lysosomal Storage Disorders (LSDs). Here, we report the discovery of a new series of non-iminosugar glucocerebrosidase inhibitors with chaperone capacity, and describe their structure–activity relationship (SAR), selectivity, cell activity and phamacokinetics.

Graphical abstract: Non-iminosugar glucocerebrosidase small molecule chaperones

Supplementary files

Article information

Article type
Concise Article
Submitted
01 Aug 2011
Accepted
21 Sep 2011
First published
24 Oct 2011

Med. Chem. Commun., 2012,3, 56-60

Non-iminosugar glucocerebrosidase small molecule chaperones

J. J. Marugan, W. Huang, O. Motabar, W. Zheng, J. Xiao, S. Patnaik, N. Southall, W. Westbroek, W. A. Lea, A. Simeonov, E. Goldin, M. A. DeBernardi and E. Sidransky, Med. Chem. Commun., 2012, 3, 56 DOI: 10.1039/C1MD00200G

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