Improving the safety characteristics of lentiviral vectors.

提高慢病毒载体的安全特性。

• 批准号: nhmrc : 349402
• 负责人: A/Pr Donald Anson
• 金额: $ 19.75万
• 依托单位: The University of Adelaide
• 依托单位国家: 澳大利亚
• 项目类别: NHMRC Project Grants
• 财政年份: 2005
• 资助国家: 澳大利亚
• 起止时间: 2005-01-01 至 2007-12-31
• 项目状态: 已结题
• 来源: https://researchdata.edu.au/improving-safety-characteristics-lentiviral-vectors/98019
• 关键词: Improving; safety; characteristics; lentiviral; vectors.

Gene therapy holds great promise for the treatment of many types of disease including inherited disorders, cancer and cardiovascular disorders. However, the potential of gene therapy has in many cases been limited by the lack of suitable technologies for gene delivery. We have developed a novel gene delivery vehicle from human immunodeficiency virus type 1 (HIV-1). Although this vehicle has many of the characteristics desired of a gene therapy vector its derivation from a retrovirus, particularly one with such an unenviable reputation, raises obvious safety concerns. In order to properly address this issue it is necessary that the vector is carefully designed and properly tested. This project aims to continue our rational, systematic and stepwise approach to the development of our vector with the aim of producing a vector that can be used with a high degree of confidence in its safety, such that it is suitable for clinical usage. Given the highly desirable properties of these vectors, and the wide range of diseases where their use is being considered, the availability of such a vector will have great significance for the widespread practical application of gene therapy. Indeed, several of the projects we are developing with our vector will in all likelihood lead to lead to clinical trials and it is clear that the conduct of these trials will depend on the availability of a suitable vector.

基因疗法在治疗包括遗传性疾病、癌症和心血管疾病在内的许多类型的疾病方面具有很大的前景。然而，基因治疗的潜力在许多情况下受到缺乏合适的基因递送技术的限制。我们已经开发了一种新的基因载体从人类免疫缺陷病毒1型（HIV-1）。尽管这种载体具有基因治疗载体所需的许多特征，但它源自逆转录病毒，特别是具有如此不令人羡慕的声誉的逆转录病毒，引起了明显的安全问题。为了适当解决这个问题，有必要仔细设计和适当测试载体。该项目旨在继续我们的合理，系统和逐步的方法来开发我们的载体，目的是生产一种可以在其安全性方面具有高度信心的载体，从而适合临床使用。考虑到这些载体的高度期望的性质，以及考虑使用它们的广泛的疾病，这种载体的可用性对于基因治疗的广泛实际应用将具有重大意义。事实上，我们正在用我们的载体开发的几个项目很可能会导致临床试验，很明显，这些试验的进行将取决于合适载体的可用性。