A New Strategy to Treat Gaucher's Disease:
Activating a Dysfunctional Enzyme

Gaucher's disease is a lysosomal storage disorder caused by a dysfunctional enzyme, acid beta-glucosidase. Recently solved structures of this enzyme give insight into the molecular basis of Gaucher's disease and the potential for treatment with isofagamine, a small molecule that increases acid β-glucosidase enzyme activity.
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Home - Introduction - Treating Gaucher's Disease - GCase Overview - GCase Structures - Summary - References

Biochemistry 462B Honors Project by Marc Bingaman (mbing@email.arizona.edu)
Dept. of Biochemistry & Molecular Biophysics, The University of Arizona, Tucson, Arizona
Instructor: Prof. Don Bourque
Last revised: April 30, 2007