ENZYME AUGMENTATION THERAPY OF GAUCHER DISEASE

戈谢病的酶增强疗法

• 批准号: 3745002
• 负责人: GREGORY A GRABOWSKI
• 金额: --
• 依托单位: CINCINNATI CHILDRENS HOSP MED CTR
• 依托单位国家: 美国
• 资助国家: 美国
• 起止时间: 至
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/3745002
• 关键词: Gaucher's disease; beta glucosidases; enzyme therapy; human subject; human therapy evaluation; inborn lysosomal enzyme disorder; metabolism disorder chemotherapy; sign /symptom; transfection

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.

本研究的目的是评估酶的功效 使用纯化的人胎盘酸β-葡糖苷酶替代 缓解1型戈谢病的症状和体征。 的 这些研究的长期目标是开发早期的方法， 和敏感性评估的治疗效果的基因为基础的 高雪氏病的治疗策略，最普遍的溶酶体 储藏病。 这些策略包括基因转移方法。