Development of a lentiviral vector for gene therapy of ADA deficiency

ADA缺乏症是一种严重的免疫缺陷，使受影响的儿童非常容易受到各种细菌和病毒的感染。骨髓移植可以纠正这种疾病，但也带来了很大的困难，特别是如果没有完全匹配的捐赠者。为此，已经开发了通过基因疗法来治愈该疾病的尝试。在最初的试验中，这是非常有希望的，但目前将ADA基因引入儿童细胞的方法可能在未来存在潜在的问题。我们现在正试图开发更安全的方法将基因携带到细胞中，在这个项目中，我们的目标是测试这些新方法是否既有效又安全。

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.