GENE THERAPY OF CANAVAN DISEASE

卡纳万病的基因治疗

• 批准号: 6265832
• 负责人: Margretta Seashore
• 金额: $ 2.58万
• 依托单位: YALE UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 1998
• 资助国家: 美国
• 起止时间: 1998-12-01 至 1999-11-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6265832
• 关键词: cerebral degeneration; child (0-11); clinical research; clinical trial phase I; gene therapy; human subject; human therapy evaluation; leukodystrophy

Treatment with a gene therapy approach for children with the childhood progressive leukodystrophy, Canavan's Disease, will be developed and characterized. This leukodystrophy results in profound psychomotor retardation and spongiform degenereration of the brain, and there is no specific treatment of the disease at present.

将开发和表征用基因治疗方法治疗患有儿童进行性脑白质营养不良（Canavan病）的儿童。 这种脑白质营养不良导致严重的精神发育迟滞和脑海绵状变性，目前尚无特异性治疗方法。