UCOE-based lentiviral vectors for effective and safe gene therapy

基于 UCOE 的慢病毒载体用于有效且安全的基因治疗

• 批准号: BB/F015526/1
• 负责人: Adrian Thrasher
• 金额: $ 76.51万
• 依托单位: University College London
• 依托单位国家: 英国
• 项目类别: Research Grant
• 财政年份: 2008
• 资助国家: 英国
• 起止时间: 2008 至 无数据
• 项目状态: 已结题
• 来源: https://gtr.ukri.org/projects?ref=BB%2FF015526%2F1
• 关键词: UCOE; based; lentiviral; vectors; effective

Gene therapy holds great promise for the cure of genetically inherited diseases including disorders of the blood such as immune system defects (severe combined immune deficiency or SCID, chronic granulomatous disease or CGD), thalassaemia, sickle cell disease, and haemophilia. This has been evidenced by the recent successful treatment by gene therapy of people with SCID and CGD. However, there are still significant challenges to ensure that the treatment works safely, and also can be used for a wider rage of conditions. The way that genes are controlled has a major impact on these issues. We have shown that it may be possible to control a therapeutic gene in a safe and effective way using a newly defined 'switch' or 'regulator' that occurs naturally in human cells. For this study we want to develop the use of this switch in gene therapy, to show that it is safe, and finally to show that model diseases can be effectively treated. If this turns out to be true, then we are hopeful that it will have a broad application to the treatment of many inherited diseases for which current treatments are inadequate.

基因疗法在治疗遗传性疾病方面有很大的希望，包括血液疾病，如免疫系统缺陷（严重联合免疫缺陷或SCID，慢性肉芽肿病或CGD），地中海贫血，镰状细胞病和血友病。这已经被最近通过基因疗法对患有SCID和CGD的人的成功治疗所证明。然而，仍然存在重大挑战，以确保治疗安全，并可用于更广泛的条件。基因控制的方式对这些问题有重大影响。我们已经证明，使用一种新定义的人类细胞中天然存在的“开关”或“调节器”，以安全有效的方式控制治疗基因是可能的。在这项研究中，我们希望开发这种开关在基因治疗中的应用，以证明它是安全的，并最终证明模型疾病可以有效治疗。如果这是真的，那么我们希望它将广泛应用于治疗目前治疗不足的许多遗传性疾病。

项目成果

Generation of functional neutrophils from a mouse model of X-linked chronic granulomatous disorder using induced pluripotent stem cells.

• DOI: 10.1371/journal.pone.0017565
• 发表时间: 2011-03-03
• 期刊: PloS one
• 影响因子: 3.7
• 作者: Mukherjee S;Santilli G;Blundell MP;Navarro S;Bueren JA;Thrasher AJ
• 通讯作者: Thrasher AJ