UCOE-based lentiviral vectors for effective and safe gene therapy

基因治疗在治疗遗传性疾病方面具有广阔的前景，包括血液疾病，例如免疫系统缺陷（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。最近通过基因疗法成功治疗 SCID 和 CGD 患者就证明了这一点。然而，要确保治疗安全有效并可用于更广泛的条件，仍然存在重大挑战。基因的控制方式对这些问题有重大影响。我们已经证明，使用人类细胞中自然发生的新定义的“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得到有效治疗。如果事实证明这是真的，那么我们希望它将广泛应用于治疗许多目前治疗方法不足的遗传性疾病。

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.