Development of gene therapy for SMEI using epilepsy model mouse.

本研究对聚乙二醇化免疫脂质体进行了研究和改进，旨在为基因治疗幼年期严重肌阵挛性癫痫提供实验依据。尽管脂质体的制备步骤已经优化，但转基因的表达不足以将治疗应用于SMEI模型小鼠。然而，本研究的结果将导致脂质体的改进，从而在靶组织中提供足够的基因表达。因此，SMEI模型小鼠的基因治疗有望在不久的将来成为可能。

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.