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
• 起止时间: 2008-09-01 至 2011-09-01
• 项目状态: 已结题
• 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; Enzyme Replacement Therapy; Genetic Disorders; Inborn Errors Of Metabolism; Protein Transduction; Urea Cycle Defects

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

尿素周期缺陷（UCD）是频繁的遗传疾病，影响多达1/8200的儿童，并且很难治疗，导致严重的神经系统疾病和早期死亡。迫切需要新颖的疗法。基因疗法是有希望的，但是李