Development of a novel hybrid rAAV/transposon gene delivery system for life-long correction of metabolic liver disease in infants and children

开发新型混合 rAAV/转座子基因传递系统，用于终身纠正婴儿和儿童代谢性肝病

• 批准号: nhmrc : 1065053
• 负责人: A/Pr Kevin Carpenter
• 金额: $ 33.73万
• 依托单位: Australian National University
• 依托单位国家: 澳大利亚
• 项目类别: Project Grants
• 财政年份: 2014
• 资助国家: 澳大利亚
• 起止时间: 2014-01-01 至 2016-12-31
• 项目状态: 已结题
• 来源: https://researchdata.edu.au/development-novel-hybrid-infants-children/455150
• 关键词: Development; novel; hybrid; rAAV; transposon

The immense potential of gene therapy for the treatment of genetic liver disease has been confirmed by recent success in a clinical trial for Haemophilia in adult males, and therapeutic benefit in other adult trials is imminent using the same technology. In the young, however, ongoing growth of the liver causes the therapeutic benefit to be short-lived. To address this problem we are developing a powerful new hybrid technology capable of conferring life-long benefit on infants and children.

基因疗法治疗遗传性肝病的巨大潜力已被最近在成年男性血友病的临床试验中取得的成功所证实，并且在其他成人试验中使用相同的技术也即将获得治疗效果。然而，在年轻人中，肝脏的持续生长导致治疗效果是短暂的。为了解决这个问题，我们正在开发一种强大的新型混合技术，能够使婴儿和儿童终身受益。