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

利用接受脊髓性肌萎缩症基因治疗的婴儿诱导的抗衣壳体液免疫来设计下一代基因转移载体

• 批准号: nhmrc : 2004320
• 金额: $ 110.6万
• 依托单位国家: 澳大利亚
• 项目类别: Ideas Grants
• 财政年份: 2021
• 资助国家: 澳大利亚
• 起止时间: 2021-01-01 至 2023-12-31
• 项目状态: 已结题
• 来源: https://researchdata.edu.au/exploiting-anti-capsid-transfer-vectors/1780326
• 关键词: Exploiting; anti; capsid; humoral; immunity

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.

经过25年的逐步进展，通过基因疗法治疗遗传性疾病的可能性已经成为治疗现实。这是通过利用基因工程使病毒无害的基因转移能力实现的。一个主要的限制是，目前高达50%的患者被排除在这些强大的工具的预先存在的免疫力之外。使用“进化在一个盘子里”，我们将工程师新一代的这些工具能够绕过预先存在的免疫隐形。