UCOE-based lentiviral vectors for effective and safe gene therapy

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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.