Exploiting anti-capsid humoral immunity induced in infants receiving gene therapy for Spinal Muscular Atrophy to engineer the next generation of gene transfer vectors

经过25年的渐进进步，基因疗法治疗遗传病的可能性已经成为治疗现实。这是通过利用基因工程使之无害的病毒的基因转移能力实现的。一个主要的限制是，目前高达50%的患者被这些强大工具的预先存在的免疫力排除在外。利用“碟子里的进化”，我们将设计出能够以秘密方式绕过先前存在的免疫的新一代工具。

After 25 years of incremental progress the possibility of treating genetic disease by gene therapy has become a therapeutic reality. This has been achieved by harnessing the gene transfer power of viruses made harmless by genetic engineering. A major limitation is that up to 50% of patients are currently excluded by pre-existing immunity to these powerful tools. Using 'evolution in a dish', we will engineer a new generation of these tools capable of bypassing pre-existing immunity by stealth.