Innovation of novel treatment and fetal therapy for ichthyosis

在本项目中，为了创新鱼鳞病的新疗法，我们制备了正常人ABCA 12基因构建体，并将该构建体转染到来自鱼鳞病患者和ABCA 12破坏的小鼠（鱼鳞病模型小鼠）的培养角质形成细胞中。我们从转染的角质形成细胞重建鱼鳞病病变，并评估转染ABCA 12基因构建体的治疗效果。此外，我们对ABCA 12破坏的小鼠的胎儿进行了治疗实验。从结果中，我们获得了线索，建立新的有效治疗先天性鱼鳞病。

In the present project, in order to innovate a novel therapy for ichhthyosis, we made a normal human ABCA12 gene construct, and performed transfection experiments of the construct into the cultured keratinocytes from ichthyosis patients and ABCA12-disrupted mice(ichthyosis model mice). We reconstructed ichthyosis lesions from the transfected keratinocytes and evaluated treatment efficacy of transfected ABCA12 gene constructs. In addition, we performed treatment experiments to fetuses of ABCA12-disrupted mice. From the results, we obtained clues to establish novel effective treatment against congenital ichthyosis.