Development of gene therapy for SMEI using epilepsy model mouse.

使用癫痫模型小鼠开发 SMEI 基因疗法。

• 批准号: 20790759
• 负责人: YAMAGATA Tetsushi
• 金额: $ 2.75万
• 依托单位: The Institute of Physical and Chemical Research
• 依托单位国家: 日本
• 项目类别: Grant-in-Aid for Young Scientists (B)
• 财政年份: 2008
• 资助国家: 日本
• 起止时间: 2008 至 2009
• 项目状态: 已结题
• 来源: https://kaken.nii.ac.jp/grant/KAKENHI-PROJECT-20790759/
• 关键词: 乳児重症ミオクロニーてんかん; 抗体修飾リポソーム; 遺伝子治療; SCN1A; 抗体修飾リボソーム; 乳児重症ミオクロ二ーてんかん

In this study, to develop gene therapy for severe myoclonic epilepsy in infancy model mice, pegylated immunoliposomes were investigated and improved. Whereas preparation steps of liposomes have been optimized, expression of transgene was not sufficient to apply the therapy to SMEI model mice. However, results of this study will lead to improvement of liposome that provides enough gene expression in the target tissue. Thus the gene therapy for SMEI model mice may be possible in near future.

在本研究中，研究并改进了聚乙二醇化免疫脂质体，以开发婴儿模型小鼠重度肌阵挛性癫痫的基因治疗方法。虽然脂质体的制备步骤已经优化，但转基因基因的表达不足以应用于SMEI模型小鼠。然而，本研究的结果将导致脂质体的改进，在靶组织中提供足够的基因表达。因此，在不久的将来，对SMEI模型小鼠进行基因治疗是可能的。