Non-invasive gene delivery for expression of therapeutic genes in oligodendrocytes: A new strategy to treat myelin diseases.

用于在少突胶质细胞中表达治疗基因的非侵入性基因递送：治疗髓磷脂疾病的新策略。

• 批准号: nhmrc : 1050277
• 负责人: A/Pr Matthias Klugmann
• 金额: $ 39.63万
• 依托单位: The University of New South Wales
• 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 2016-12-31
• 项目状态: 已结题
• 来源: https://researchdata.edu.au/non-invasive-gene-myelin-diseases/112148
• 关键词: Non; invasive; gene; delivery; expression

White matter diseases are debilitating childhood disorders caused by defects in the insulating myelin sheath normally covering and protecting the nerve fibres from damage. There is currently no effective treatment but the delivery of a genetic medicine to the diseased myelin forming cells in the brain could be curative. This project aims at establishing the safe, efficient and non-invasive delivery of therapeutic genes to myelin forming cells as a gene therapy for white matter disorders.

白质疾病是一种衰弱的儿童疾病，由绝缘髓鞘缺陷引起，通常覆盖和保护神经纤维免受损害。目前还没有有效的治疗方法，但向大脑中患病的髓鞘形成细胞输送基因药物可能是治愈的。该项目旨在建立安全、高效和非侵入性的治疗性基因输送到髓鞘形成细胞作为白质疾病的基因治疗。