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Invention E-257-2010/0

Glucocerebrosidase Activators as a Treatment for Gaucher Disease

Lead Inventor: Juan Marugan (NCATS)
Inventors: Ehud Goldin (NHGRI), Omid Motabar (NHGRI), Samarjit Patnaik (NCATS), Ellen Sidransky (NHGRI), Noel Southall (NCATS), Wendy Westbroek (NHGRI), Wei Zheng (NCATS)
Ref. No.: E-257-2010/0

Abstract: This technology is a collection of small molecule activators of a genetically defective version of the enzyme called glucocerebrosidase (GCase), which causes Gaucher disease.

Publications:

Discovery, Structure-Activity Relationship, and Biological Evaluation of Noninhibitory Small Molecule Chaperones of GlucocerebrosidaseJournal of Medicinal Chemistry • June 28, 2012 • Probe Development Branch, NCATS Chemical Genomics Center

New Non-Iminosugar Glucocerebrosidase Chaperone SeriesMedChemComm • January 2012 • Probe Development Branch, NCATS Chemical Genomics Center

A High Throughput Glucocerebrosidase Assay Using the Natural Substrate GlucosylceramideAnalytical and Bioanalytical Chemistry • January 2012 • Probe Development Branch, NCATS Chemical Genomics Center

Evaluation of Quinazoline Analogues as Glucocerebrosidase Inhibitors with Chaperone ActivityJournal of Medicinal Chemistry • Feb. 24, 2011 • Probe Development Branch, NCATS Chemical Genomics Center

Last updated: 07-21-2016
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