Analysis and manipulation of the genome-wide integration signatures of gamma-retroviral and lentiviral vectors in human haematopoietic stem cells

人类造血干细胞中γ-逆转录病毒和慢病毒载体的全基因组整合特征的分析和操作

• 批准号: nhmrc : 1026710
• 负责人: Prof Ian Alexander
• 金额: $ 40.82万
• 依托单位: The University of Sydney
• 依托单位国家: 澳大利亚
• 项目类别: Project Grants
• 财政年份: 2012
• 资助国家: 澳大利亚
• 起止时间: 2012-01-01 至 2014-12-31
• 项目状态: 已结题
• 来源: https://researchdata.edu.au/analysis-manipulation-genome-stem-cells/110514
• 关键词: Analysis; manipulation; genome; wide; integration

Gene therapy has been successful in treating several diseases involving the bone marrow, but has been associated with the development of leukaemia in a number of patients. The cause has been tracked to the gene transfer technology used and associated damage to the genetic blueprint of treated cells. In this study we plan to use high-throughput genetic analysis to better understand the nature of this damage and to develop strategies to improve the safety of the gene repair process.

基因疗法已成功地治疗了几种涉及骨髓的疾病，但与许多患者的白血病的发展有关。其原因已被追踪到所使用的基因转移技术和对处理细胞的遗传蓝图的相关损害。在这项研究中，我们计划使用高通量遗传分析来更好地了解这种损伤的性质，并制定策略来提高基因修复过程的安全性。