The objective of this research is to evaluate the efficacy of enzyme replacement using purified human placental acid beta-glucosidase for the alleviation of the symptoms and signs of Gaucher disease type 1. The long-term objective of these studies is to develop methods for the early and sensitive assessment of therapeutic efficacy of gene-based therapeutic strategies for Gaucher disease, the most prevalent lysosomal storage disease. These strategies include gene transfer approaches.
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