Harnessing RNA interference in gene therapy vectors for β-thalassaemia

利用 RNA 干扰基因治疗载体治疗 β 地中海贫血

• 批准号: nhmrc : GNT1147867
• 负责人: A/Pr Saovaros Svasti
• 金额: $ 70.93万
• 依托单位: Monash University
• 依托单位国家: 澳大利亚
• 项目类别: Project Grants
• 财政年份: 2018
• 资助国家: 澳大利亚
• 起止时间: 2018-01-01 至 无数据
• 项目状态: 已结题
• 来源: https://researchdata.edu.au/harnessing-rna-interference-vectors-thalassaemia/1348853
• 关键词: Harnessing; RNA; interference; gene; therapy

There is an urgent need to develop safe and effective treatments for β-thalassaemia. We anticipate that α-globin-specific RNAi sequences will synergise with β-globin transgene expression to achieve balanced α-/β-globin ratio in a clinical setting. Given that one of the major issues with current gene therapy vectors is achieving high levels of expression, we believe this will be a more effective gene therapy strategy than β-globin transgene expression alone.

迫切需要开发安全有效的β-地中海贫血治疗方法，我们预计α-珠蛋白特异性RNAi序列将与β-珠蛋白转基因表达协同作用，以在临床环境中实现平衡的α-/β-珠蛋白比例。当前基因治疗载体的主要问题之一是实现高水平表达，我们相信这将是比单独的β-珠蛋白转基因表达更有效的基因治疗策略。