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GAUCHER'S DISEASE--BIOCHEMICAL AND CLINICAL STUDIES

GAUCHER'S DISEASE--BIOCHEMICAL AND CLINICAL STUDIES
戈谢氏病——生化和临床研究
批准号：
3922532
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/3922532
关键词：
Gaucher's disease chemical structure function child (0-11) developmental genetics enzyme linked immunosorbent assay enzyme mechanism enzyme structure enzyme substrate enzyme therapy gene expression gene therapy glucosylceramidase human subject human tissue immunochemistry inborn lysosomal enzyme disorder inborn metabolism disorder diagnosis metabolism disorder chemotherapy molecular genetics monoclonal antibody neurochemistry pathologic process radioimmunoassay tissue /cell culture

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中文摘要
翻译
高雪病的治疗依赖于临床和基础 对这种疾病的科学知识。病人已经被 进行了广泛的研究，并发现了并发症。研究与实践 葡萄糖脑苷酶涉及生物化学、细胞生物学和 酶的分子遗传学。阿司匹林的药效学评价 甘露糖终止的人胎盘葡萄糖脑苷酶 完成。在这项研究中获得的结果将构成 该酶对高雪病患者的疗效观察 针对的是储存脂质的细胞。
英文摘要
Therapy for Gaucher's disease depends upon board clinical and basic scientific knowledge of the disorder. Patients have been extensively studied and complications identified. Research on glucocerebrosidase addresses the biochemistry, cell biology, and molecular genetics of the enzyme. A pharmacodynamic assessment of mannose-terminated human placental glucocerebrosidase is nearly completed. Results obtained in this study will form the basis of an efficacy investigation in Gaucher patients with this enzyme that is targeted to lipid storing cells.
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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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