ENZYME AUGMENTATION THERAPY OF GAUCHER DISEASE

这项研究的目的是评价酶的疗效。 用纯化的人胎盘酸β-葡萄糖苷酶替代 缓解高谢病1型的症状和体征。 这些研究的长期目标是开发早期的方法 以基因为基础的疗效敏感评估 最常见的溶酶体型高谢病的治疗策略 储藏病。这些策略包括基因转移方法。

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.