Functional restoration of OTC deficient primary human hepatocytes in a xenograft model using an AAV vector uniquely configured for impending clinical trial use.

使用专门配置用于即将进行的临床试验的 AAV 载体，在异种移植模型中恢复 OTC 缺陷的原代人肝细胞的功能。

• 批准号: nhmrc : 1080333
• 负责人: Prof Ian Alexander
• 金额: $ 15.7万
• 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: 2015
• 资助国家: 澳大利亚
• 起止时间: 2015-01-01 至 2016-12-31
• 项目状态: 已结题
• 来源: https://researchdata.edu.au/functional-restoration-otc-trial-use/518740
• 关键词: Functional; restoration; OTC; deficient; primary

The aim of this project is to acquire preclinical data which will underpin an international gene therapy trial for severe ornithine transcarbamylase (OTC) deficiency, the most prevalent urea cycle defect in infants and children. In most severe cases, liver transplantation is required for long term survival. We, with colleagues at Stanford University, have recently developed a novel gene therapy tool for optimal targeting of human liver cells which will be tested in a humanised mouse model.

该项目的目的是获得临床前数据，这些数据将支持一项针对严重鸟氨酸转氨酶(OTC)缺乏症的国际基因治疗试验，OTC缺乏症是婴儿和儿童中最常见的尿素循环缺陷。在最严重的情况下，需要肝移植才能长期存活。我们和斯坦福大学的同事们最近开发了一种新的基因治疗工具，用于优化人类肝细胞的靶向，将在人源化的小鼠模型中进行测试。