课题基金 / 基金详情

GENETIC THERAPY OF FABRY DISEASE

GENETIC THERAPY OF FABRY DISEASE
法布里病的基因治疗
批准号：
2579673
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/2579673
关键词：
Fabry's disease Retroviridae alpha galactosidase clinical research clinical trial phase I complementary DNA enzyme activity gene therapy genetic transduction hematopoietic stem cells human genetic material tag human subject human therapy evaluation inborn lysosomal enzyme disorder inborn metabolism disorder sex linked trait transfection /expression vector

项目摘要

项目成果

R O BRADY的其他基金

相关文献

中文摘要
翻译
法布里病是一种x连锁遗传隐性代谢疾病
英文摘要
Fabry disease is an X-linked inherited recessive metabolic disorder caused by insufficient activity of the lysosomal enzyme alpha- galactosidase A (ceramidetrihexosidase). In contrast with the remarkable benefit of enzyme replacement therapy developed by DMNB for patients with Gaucher disease, little progress has been made concerning specific treatment for patients with Fabry disease. We propose to develop gene replacement therapy for patients with this metabolic disorder. Retroviral vectors containing the cDNA of human alpha-galactosidase A will be produced, and the efficiency of transduction of patients' hematopoietic stem cells will be determined. When a vector with sufficient activity and titer is available, we shall develop a Phase l gene therapy trial for patients with Fabry disease.
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 AN ANALOGUE OF HUMAN GM1 GANGLIOSIDOSIS