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 phamacokinetics.

SUBMITTER: Marugan JJ 

PROVIDER: S-EPMC3351140 | biostudies-literature | 2012 Jan

REPOSITORIES: biostudies-literature