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ENZYME REPLACEMENT THERAPY IN DISORDERS THAT AFFECT THE CENTRAL NERVOUS SYSTEM

ENZYME REPLACEMENT THERAPY IN DISORDERS THAT AFFECT THE CENTRAL NERVOUS SYSTEM
影响中枢神经系统疾病的酶替代疗法
批准号：
2579614
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/2579614
关键词：
Gaucher's disease Mammalia beta galactosidase brain central nervous system drug delivery systems drug metabolism drug receptors enzyme therapy gangliosides gangliosidosis GM1 glucosylceramidase inborn metabolism disorder injection /infusion metabolism disorder chemotherapy neurons nonhuman therapy evaluation placenta tissue /cell culture

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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 are now developing procedures to deliver useful amounts of enzymes to the brain in patients with hereditary metabolic storage disorders. We have examined 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. We are characterizing the receptors involved in the uptake of glucocerebrosidase and other enzymes by neurons in tissue culture.
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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 AN ANALOGUE OF HUMAN GM1 GANGLIOSIDOSIS
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