Development of lentiviral vectors for the treatment of X-linked severe combined immunodeficiency (SCID-X1)

第一个成功的基因治疗临床试验于2000年报道，用于治疗x连锁严重联合免疫缺陷（SCID-X1），通常被称为“泡泡男孩”疾病。11例患者中有3例随后发展为白血病，这促使人们需要开发替代载体来传递基因，例如基于hiv -1的慢病毒载体。本项目将评估慢病毒载体在体内的有效性和安全性，以及它们治疗SCID-X1的治疗潜力。

The first successful gene therapy clinical trial was reported in 2000 with the treatment of X-linked severe combined immunodeficiency (SCID-X1), commonly known as “bubble-boy” disease. The subsequent development of leukaemia in 3 of 11 patients has prompted the need to develop alternative vectors for gene delivery, such as HIV-1-based lentiviral vectors. This project will evaluate the efficacy and safety of lentiviral vectors in vivo, and hence their therapeutic potential for treating SCID-X1.