Development of an enhanced lentiviral vector for gene therapy of ADA-SCID

腺苷脱氨酶缺乏症是一种受影响的儿童缺乏功能免疫系统并在出生第一年死于感染的情况。基因缺陷的性质是这样的，身体的其他系统也发现了严重的缺陷。由于其他治疗方法的局限性，基因治疗最近在纠正免疫缺陷方面取得了一些成功。在这项研究中，我们希望以此为基础，测试更好的基因治疗递送载体，以纠正免疫系统和非免疫系统问题。我们还将使用与当前使用的构造相比具有更高安全性的载体。我们将在细胞系统中测试我们的新载体，并致力于纠正这种疾病的小鼠模型。

Adenosine deaminase deficiency is a condition in which affected children lack a functional immune system and die from infection in the first year of life. The nature of the gene defect is such that severe defects are also found in other systems of the body. Due to the limitations of other treatment options, gene therapy has recently been used with some success in correcting the immune defects. In this study we hope to build on this and test better gene therapy delivery vectors that may correct both the immune system and non-immune system problems. We will also use vectors that have improved safety profiles compared to presently used constructs. We will test our new vectors in cellular systems and also aim to correct a murine model of the disease.