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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：
3881725
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/3881725
关键词：
Gaucher's disease clinical trials congenital nervous system disorder developmental neurobiology enzyme induction /repression enzyme mechanism enzyme structure glycolipids human subject human therapy evaluation mannose medical complication molecular genetics placental hormones

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中文摘要
翻译
摘要：高谢病的成功治疗有赖于 全面了解该病的临床和基本科学知识。 对患者进行了广泛的研究，并确定了并发症。 葡萄糖脑苷酶的研究涉及生物化学、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已经完成。 用酶已经取得了非常令人满意的结果 高谢病患者的替代疗法。
英文摘要
Summary: Successful therapy for Gaucher's disease depends upon a comprehensive 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 has been completed. Extraordinarily gratifying results have been obtained with enzyme replacement therapy in patients with Gaucher's disease.
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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 AN ANALOGUE OF HUMAN GM1 GANGLIOSIDOSIS
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