Development of a lentiviral vector for gene therapy of ADA deficiency

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ADA deficiency is a severe form of immunodeficiency which leaves affected children very vulnerable to infections from all types of bacteria and viruses. Bone marrow transplant can correct the disease but carries with it major difficulties especially if a fuly matched donor is not available. For this reason, attempts to cure the disease by gene therapy have been developed. In initial trials, this has been very promising but the current methods by which the ADA gene is introduced into the child s cells may have potential problems for the future. We are now trying to develop safer ways of carrying genes into cells and in this project we aim to test whether these new methods are both effective and safe.