Development epigenomic restoration therapy for autistic disorders

表观遗传学是一种可逆机制。因此，我们研究了具有代表性的自闭症Rett综合征的表观基因组恢复。我们最初计划开发一种化学物质，以一种基因特异性的方式恢复表观基因组状态。然而，我们意识到需要表观基因组修复的基因相对较多。因此，我们寻找具有全球效应的化学物质。因此，我们证明了一种治疗Rett综合征癫痫的药物具有全球效应，可以上调许多基因。此外，我们发现了更有效的新化学物质，这表明当我们在新建立的患者来源的iPS细胞中证实其效果时，这些化学物质将成为治疗Rett综合征的新候选药物。

Epigenetics is a reversible mechanism. Thus, we investigated epigenomic restoration for a representative autistic disorder, Rett syndrome. We initially planned to develop a chemical to restore epigenomic status in a gene-specific manner. However, we realized that genes that require epigenomic restoration were relatively many. Therefore, we searched chemicals with a global effect. As a result, we demonstrated that a drug for epilepsy of Rett syndrome had a global effect with up-regulation of many genes. Furthermore, we identified more effective new chemicals, suggesting that these will be new drug candidates for Rett syndrome when we confirm the effect in our newly established patients-derived iPS cells.