Targeting the mitochondrial transcriptome to treat mitochondrial disease

靶向线粒体转录组治疗线粒体疾病

• 批准号: nhmrc : 1045677
• 负责人: A/Pr Aleksandra Filipovska
• 金额: $ 30.46万
• 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: 2013
• 资助国家: 澳大利亚
• 起止时间: 2013-01-01 至 2015-12-31
• 项目状态: 已结题
• 来源: https://researchdata.edu.au/targeting-mitochondrial-transcriptome-mitochondrial-disease/111779
• 关键词: Targeting; mitochondrial; transcriptome; treat; disease

Mitochondrial diseases are a diverse group of progressive and debilitating disorders for which there are no effective treatments. Furthermore it is currently impossible to apply gene therapy or RNA interference approaches to understand how mitochondria function or to treat mitochondrial diseases. We are developing a new technology that makes it possible to rationally manipulate mammalian mitochondrial RNAs for the first time, providing a unique approach for mitochondrial disease therapies.

线粒体疾病是一组多样的进行性和衰弱性疾病，对其没有有效的治疗方法。此外，目前不可能应用基因治疗或RNA干扰方法来了解线粒体如何发挥功能或治疗线粒体疾病。我们正在开发一种新技术，首次能够合理地操纵哺乳动物线粒体RNA，为线粒体疾病治疗提供了一种独特的方法。