Development of a lentiviral vector for gene therapy of ADA deficiency

开发用于 ADA 缺陷基因治疗的慢病毒载体

• 批准号: G0802483/1
• 负责人: Hubert Gaspar
• 金额: $ 71.97万
• 依托单位: University College London
• 依托单位国家: 英国
• 项目类别: Research Grant
• 财政年份: 2010
• 资助国家: 英国
• 起止时间: 2010 至 无数据
• 项目状态: 已结题
• 来源: https://gtr.ukri.org/projects?ref=G0802483%2F1
• 关键词: Development; lentiviral; vector; gene; therapy

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.

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。骨髓移植可以治愈这种疾病，但也有很大的困难，尤其是在找不到完全匹配的供体的情况下。因此，人们开始尝试通过基因疗法来治愈这种疾病。在最初的试验中，这很有希望，但目前将ADA基因引入儿童细胞的方法可能会在未来产生潜在的问题。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。