Enhancing ocular gene therapy using glycosidic enzymes

使用糖苷酶增强眼部基因治疗

• 批准号: G1000268/1
• 负责人: Jasmina Cehajic-Kapetanovic
• 金额: $ 28.14万
• 依托单位: University of Manchester
• 依托单位国家: 英国
• 项目类别: Fellowship
• 财政年份: 2010
• 资助国家: 英国
• 起止时间: 2010 至 无数据
• 项目状态: 已结题
• 来源: https://gtr.ukri.org/projects?ref=G1000268%2F1
• 关键词: Enhancing; ocular; gene; therapy; using

Gene therapy shows great promise in treating human blindness due to genetic diseases affecting the retina. Healthy genes, contained in modified viruses, are delivered by injection under the retina, a thin later of tissue that converts light into signals that are sent to our brain. However, this approach can potentially damage the retina and cannot deliver the genes across the entire retina. A potentially safer and more effective approach would be to inject the gene-carrying viruses into the vitreous, a gel-like structure in front of the retina. However, to date this approach has not worked very well with only a small number of retinal cells receiving the gene. My research to date has shown that if we use enzymes in combination with the viruses we achieve much better gene delivery to the retina following injection into the vitreous. The aim of this project is to optimise the intravitreal combination of virus and enzymes to deliver genes right across the retina. I will then see if I can demonstrate recovery of retinal function in a mouse that, because of genetic alterations, has no vision. This may ultimately lead to exciting advancements in treating patients with a variety of retinal diseases.

基因疗法在治疗因影响视网膜的遗传性疾病引起的人类失明方面显示出巨大的希望。健康的基因，包含在修改后的病毒中，通过注射到视网膜下来传递，视网膜是一层薄的组织，可以将光转化为发送到我们大脑的信号。然而，这种方法可能会损害视网膜，并且无法将基因传递到整个视网膜。一种潜在的更安全和更有效的方法是将携带基因的病毒注射到玻璃体中，玻璃体是视网膜前面的凝胶状结构。然而，到目前为止，这种方法还没有很好地工作，只有少数视网膜细胞接受基因。迄今为止，我的研究表明，如果我们将酶与病毒结合使用，我们可以在注射到玻璃体后更好地将基因传递到视网膜。该项目的目的是优化病毒和酶的玻璃体内组合，以将基因直接传递到视网膜。然后，我将看看我是否能证明视网膜功能的恢复在一只老鼠，因为基因改变，没有视力。这可能最终导致治疗各种视网膜疾病患者的令人兴奋的进步。