Clinical trial of self-inactivating vectors for gene therapy of X-linked Severe Combined Immunodeficiency (SCID-X1)

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Gene therapy has the potential to treat a wide range of inherited diseases. Recently, several trials have shown that children with defects of their immune system (often called bubble babies because they are kept in germ free rooms) can be successfully treated in this way by putting new genes into the blood-making factory called bone marrow. However, the way in which the new genes are transplanted into the bone marrow, using modified viruses or vectors, has also been shown in a few children to have unfortunate side-effects. We now know that these arise because the vectors inadvertantly activate other genes that keep the cells in our body healthy. In the worst case, this can cause cancer. However, we also now believe that the risk of this occuring can be substantially limited by improved design of the vectors themselves. Following on from highly encouraging experiments in the laboratory, we would now like to test this strategy in patients for whom coventional bone marrow transplantation is difficult. This will not only provide a new treatment for patients with immune defects, but will provide a platform on which gene therapy can be safely developed for many other diseases.