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

对于患有严重肝病的患者来说，肝移植往往是唯一的治疗选择，而且供体器官短缺，以及需要终身药物疗法来防止排斥反应，这让移植变得复杂起来。通过基因疗法修复患者自己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，对患病肝细胞中的遗传拼写错误进行精确纠正，而不需要首先将它们从体内移除。

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.