In vivo gene transfer and phenotype correction of normal and urea-cycle deficient primary human hepatocytes in chimeric mouse-human livers: Towards gene therapy for metabolic liver disease

嵌合小鼠-人肝脏中正常和尿素循环缺陷的原代人肝细胞的体内基因转移和表型校正：迈向代谢性肝病的基因治疗

• 批准号: nhmrc : 1008021
• 负责人: A/Pr Filip Braet
• 金额: $ 32.92万
• 依托单位: The University of Sydney
• 依托单位国家: 澳大利亚
• 项目类别: NHMRC Project Grants
• 财政年份: 2011
• 资助国家: 澳大利亚
• 起止时间: 2011-01-01 至 2013-12-31
• 项目状态: 已结题
• 来源: https://researchdata.edu.au/vivo-gene-transfer-liver-disease/109268
• 关键词: vivo; gene; transfer; phenotype; correction

Genetic liver disease imposes a major health and economic burden. Existing medical treatments are frequently inadequate, often necessitating liver transplantation which carries its own limitations and risks. Using a gene therapy approach we have achieved life-long cure of mice with OTC-deficiency, a condition with a high risk of disability and death in affected infants. This application focuses on translating this success in mice through to human therapy.

遗传性肝病造成了重大的健康和经济负担。现有的医学治疗往往是不够的，往往需要肝移植，这带来了自己的局限性和风险。使用基因治疗方法，我们已经实现了OTC缺乏症小鼠的终身治愈，这种疾病在受影响的婴儿中具有高残疾和死亡风险。该应用程序的重点是将小鼠的成功转化为人类治疗。