Directed evolution of AAV capsid variants for enhanced targeted genome editing in the human liver

AAV 衣壳变体的定向进化可增强人类肝脏中的靶向基因组编辑

• 批准号: nhmrc : GNT1108311
• 负责人: Dr Leszek Lisowski
• 金额: $ 38.6万
• 依托单位国家: 澳大利亚
• 项目类别: Project Grants
• 财政年份: 2016
• 资助国家: 澳大利亚
• 起止时间: 2016-01-01 至 无数据
• 项目状态: 已结题
• 来源: https://researchdata.edu.au/directed-evolution-aav-human-liver/1346639
• 关键词: Directed; evolution; AAV; capsid; variants

Liver transplantation is often the only treatment option available for patients with severe liver disease, and is complicated by a shortage of donor organs and the need for life-long drug therapy to prevent rejection. Repair of a patient’s own liver by gene therapy is a promising alternative. This project focuses on developing the technology required to undertake precise correction of genetic spelling errors in diseased liver cells without the need to first remove them from the body.

肝移植通常是严重肝病患者的唯一治疗选择，并且由于供体器官短缺以及需要终身药物治疗以防止排斥反应而变得复杂。通过基因疗法修复患者自身的肝脏是一种有前途的替代方案。该项目的重点是开发精确纠正患病肝细胞中遗传拼写错误所需的技术，而无需首先将其从体内取出。