Hepatocellular Carcinoma: understanding the genotoxic risks of liver-targeted gene therapy using recombinant AAV vectors

使用称为AAV的工程病毒的基因转移技术的进展支持了血友病B的治疗成功，并提供了治疗许多其他肝脏疾病的令人兴奋的前景。虽然继续提高基因转移效率至关重要，但同样需要关注安全性。我们在AAV中发现了一个遗传元件，我们认为这是解锁基于AAV的基因转移技术安全性的准确分析的关键。在这里，我们建议转动钥匙。

Advances in gene transfer technology using an engineered virus known as AAV underpin success in the treatment of haemophila B, and offer the exciting prospect of treating many other liver diseases. While continued improvement of gene transfer efficiency is essential there is an equal need to focus on safety. We have discovered a genetic element in AAV that we believe is a key to unlocking accurate analysis of the safety of AAV-based gene transfer technology. Here we propose to turn the key.