ENZYME REPLACEMENT THERAPY IN AN ANALOGUE OF HUMAN GM1 GANGLIOSIDOSIS

类人 GM1 神经节节病的酶替代疗法

• 批准号: 3760329
• 负责人: R O BRADY
• 金额: --
• 依托单位: NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE
• 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/3760329
• 关键词: Gaucher's disease; Mammalia; beta galactosidase; brain; drug delivery systems; enzyme therapy; gangliosidosis GM1; glucosylceramidase; nonhuman therapy evaluation; placenta

Enzyme replacement therapy has been shown to be extraordinarily effective for patients with Type 1 (non-neuronopathic) Gaucher's disease. We now need to develop procedures to deliver useful amounts of enzymes to the brain in patients with hereditary metabolic storage disorders. We are examining the effect of human placental beta-galactosidase on the amount of ganglioside GM1 in animal analogues of human generalized (GM1) gangliosidosis using a new intracerebral protein delivery system.We are also determining the distribution of glucocerebrosidase in the brain using convection-enhanced intracerebral injection of this enzyme.

酶替代疗法已被证明是非常有效的 1型（非神经病性）戈谢病患者。 我们现在 需要开发程序，以提供有用的酶量， 遗传性代谢储存障碍患者的大脑。 我们 检测人胎盘β-半乳糖苷酶对人胎盘β-半乳糖苷酶的量的影响 神经节苷脂GM 1在人广义神经节苷脂（GM 1）动物类似物中的作用 神经节苷脂沉积症使用一种新的脑内蛋白质输送系统。 还确定了葡萄糖脑苷脂酶在大脑中的分布 使用对流增强的这种酶的脑内注射。