Synthesis and testing of isofagomine and derivatives as inhibitors of human glucocerebrosidase
Gaucher disease is a lysosomal storage disorder in which the activity of the enzyme glucocerebrosidase (GCase) is deficient. As a result, the substrate of GCase, glucosylceramide, accumulates in lysosomes of Gaucher patients, causing enlargement of the spleen and liver, bone deformity and neurode...
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| Language: | English |
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University of British Columbia
2009
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| Online Access: | http://hdl.handle.net/2429/7568 |