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 : 1108311
• 负责人: Prof Ian Alexander
• 金额: $ 26.47万
• 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: 2016
• 资助国家: 澳大利亚
• 起止时间: 2016-01-01 至 2018-12-31
• 项目状态: 已结题
• 来源: https://researchdata.edu.au/directed-evolution-aav-human-liver/663037
• 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唯一可用的治疗选择，并且由于供体器官短缺和需要终身药物治疗以防止排斥反应而复杂化。通过基因疗法修复病人自身的肝脏是一个很有前途的选择。该项目的重点是开发所需的技术，以精确纠正患病肝细胞中的基因拼写错误，而不需要首先将它们从体内移除。