The L-type calcium channel as a reporter of successful morpholino oligomer therapy in treatment of Duchenne Muscular Dystrophy cardiomyopathy

L型钙通道作为吗啉低聚物治疗杜氏肌营养不良症心肌病成功的报告者

• 批准号: nhmrc : 1062740
• 负责人: A/Pr Livia Hool
• 金额: $ 39.68万
• 依托单位: The University of Western Australia
• 依托单位国家: 澳大利亚
• 项目类别: Project Grants
• 财政年份: 2014
• 资助国家: 澳大利亚
• 起止时间: 2014-01-01 至 2016-12-31
• 项目状态: 已结题
• 来源: https://researchdata.edu.au/l-type-calcium-dystrophy-cardiomyopathy/455017
• 关键词: type; calcium; channel; reporter; successful

Duchenne Muscular Dystrophy is a fatal muscle wasting disorder. We have previously characterised how the heart fails in a mouse model of muscular dystrophy. We now have preliminary data demonstrating that treatment of mice with morpholino oligomers can rescue cardiac function. This project will fully characterise the effect of the treatment on heart function and optimise therapy regimes with the view to utilising the optimised protocol as a guideline in treating cardiomyopathy in Duchenne Muscular Dystrophy boys.

杜氏肌营养不良症是一种致命的肌肉萎缩性疾病。我们之前已经描述了肌肉萎缩症小鼠模型中心脏如何衰竭。我们现在有初步的数据表明，用吗啉代寡聚体治疗小鼠可以挽救心脏功能。该项目将充分验证治疗对心脏功能的影响，并优化治疗方案，以期利用优化的方案作为治疗杜氏肌营养不良症男孩心肌病的指南。