Journal
JOURNAL OF MEDICINAL CHEMISTRY
Volume 55, Issue 12, Pages 5734-5748Publisher
AMER CHEMICAL SOC
DOI: 10.1021/jm300063b
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Funding
- Molecular Libraries Initiative of the NIH Roadmap for Medical Research
- Intramural Research Program of the National Human Genome Research Institute, National Institutes of Health
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A major challenge in the field of Gaucher disease has been the development of new therapeutic strategies including molecular chaperones. All previously described chaperones of glucocerebrosidase are enzyme inhibitors, which complicates their clinical development because their chaperone activity must be balanced against the functional inhibition of the enzyme. Using a novel high throughput screening methodology, we identified a chemical series that does not inhibit the enzyme but can still facilitate its translocation to the lysosome as measured by immunostaining of glucocerebrosidase in patient fibroblasts. These compounds provide the basis for the development of a novel approach toward small molecule treatment for patients with Gaucher disease.
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