Reversal of diabetes in a humanised mouse using a clinically applicable vector system

使用临床适用的载体系统逆转人源化小鼠的糖尿病

• 批准号: nhmrc : 1086256
• 负责人: A/Pr Bronwyn O'Brien
• 金额: $ 56.16万
• 依托单位: University of Technology Sydney
• 依托单位国家: 澳大利亚
• 项目类别: Project Grants
• 财政年份: 2015
• 资助国家: 澳大利亚
• 起止时间: 2015-01-01 至 2018-12-31
• 项目状态: 已结题
• 来源: https://researchdata.edu.au/reversal-diabetes-humanised-applicable-vector/519125
• 关键词: Reversal; diabetes; humanised; mouse; using

Somatic gene therapy is one of the strategies that is being considered to cure Type I diabetes. Specifically, we wish to engineer liver cells to replace beta cell function. The aim of this project is to design a clinically-applicable protocol for the reversal of diabetes using a recombinant adeno-associated vector that delivers genes to human livers with high efficiency showing long term expression without pathogenicity and immunogenicity following a simple intra-peritoneal injection.

体细胞基因治疗是正在考虑治愈 I 型糖尿病的策略之一。具体来说，我们希望改造肝细胞来取代β细胞的功能。该项目的目的是设计一种临床适用的方案，使用重组腺相关载体将基因高效地输送到人类肝脏，在简单的腹膜内注射后显示出长期表达，无致病性和免疫原性。