课题基金 / 基金详情

ENZYME REPLACEMENT THERAPY IN AN ANALOGUE OF HUMAN GM1 GANGLIOSIDOSIS

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：
$0.0万
依托单位：
NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE
依托单位国家：
美国
项目类别：
财政年份：
--
资助国家：
美国
项目状态：
未结题
起止时间：
至
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

项目摘要

项目成果

R O BRADY的其他基金

相似基金

相关文献

中文摘要
翻译
酶替代疗法已被证明非常有效
英文摘要
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.
期刊论文(0)
专著(0)
科研奖励(0)
会议论文
ENZYME REPLACEMENT THERAPY IN DISORDERS THAT AFFECT THE CENTRAL NERVOUS SYSTEM
GENE THERAPY OF INHERITED ENZYME DEFICIENCIES
ENZYME REPLACEMENT THERAPY IN AN ANALOGUE OF HUMAN GM1 GANGLIOSIDOSIS
ENZYME REPLACEMENT THERAPY IN DISORDERS THAT AFFECT THE CENTRAL NERVOUS SYSTEM
海外基金