SMN immunogene therapy for motor neuron disorders

SMN 免疫基因治疗运动神经元疾病

• 批准号: nhmrc : GNT1104299
• 负责人: A/Pr Bradley Turner
• 金额: $ 59.2万
• 依托单位国家: 澳大利亚
• 项目类别: Project Grants
• 财政年份: 2016
• 资助国家: 澳大利亚
• 起止时间: 2016-01-01 至 无数据
• 项目状态: 已结题
• 来源: https://researchdata.edu.au/smn-immunogene-therapy-neuron-disorders/1346349
• 关键词: SMN; immunogene; therapy; motor; neuron

We have designed novel gene therapy agents to treat MND. This therapeutic approach uniquely combines gene therapy agents with antibodies which stimulate motor neuron health and connections to muscle. This project will comprehensively characterise the therapeutic effects of our novel gene therapy agents in MND mice. We predict that our gene therapy complexes will improve motor neuron survival,motor function and lifespan in MND mice.

我们设计了新型基因治疗药物来治疗MND。这种治疗方法独特地将基因治疗剂与刺激运动神经元健康和与肌肉连接的抗体相结合。本项目将全面验证我们的新型基因治疗剂对MND小鼠的治疗效果。我们预测，我们的基因治疗复合物将改善MND小鼠的运动神经元存活、运动功能和寿命。