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

开发用于治疗X连锁严重联合免疫缺陷（SCID-X1）的慢病毒载体

• 批准号: nhmrc : 477112
• 负责人: Dr Claire Deakin
• 金额: $ 4.76万
• 依托单位: Children's Medical Research Institute
• 依托单位国家: 澳大利亚
• 项目类别: NHMRC Postgraduate Scholarships
• 财政年份: 2008
• 资助国家: 澳大利亚
• 起止时间: 2008-01-01 至 2011-12-31
• 项目状态: 已结题
• 来源: https://researchdata.edu.au/development-lentiviral-vectors-scid-x1/115381
• 关键词: Development; lentiviral; vectors; treatment; linked

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.

第一个成功的基因治疗临床试验是在2000年报道的，用于治疗X连锁严重联合免疫缺陷（SCID-X1），俗称“泡泡男孩”病。11名患者中有3名随后发生白血病，这促使需要开发用于基因递送的替代载体，例如基于HIV-1的慢病毒载体。该项目将评估慢病毒载体在体内的有效性和安全性，从而评估其治疗SCID-X1的治疗潜力。