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Development of Enzyme Replacement Therapy for Urea Cycle Defects

Development of Enzyme Replacement Therapy for Urea Cycle Defects
针对尿素循环缺陷的酶替代疗法的开发
批准号：
173692
负责人：
Campeau Philippe M
金额：
$13.66万
依托单位：
Baylor College of Medicine (Texas)
依托单位国家：
加拿大
项目类别：
Fellowship Programs
财政年份：
2008
资助国家：
加拿大
项目状态：
已结题
起止时间：
2008-09-01 至 2011-09-01
来源：
https://webapps.cihr-irsc.gc.ca/decisions/p/project_details.html?applId=173692&lang=en
关键词：
Enzyme Replacement Therapy Genetic Disorders Inborn Errors Of Metabolism Protein Transduction Urea Cycle Defects

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Urea cycle defects (UCDs) are frequent genetic disorders, affecting up to 1/8200 children, and are notably difficult to treat, causing severe neurologic disease and early death. Novel therapies are urgently needed. Gene therapy is promising, but issues li
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