Study on the mechanism of aggregate formation in neurodegeneration

本项目主要研究聚集体的形成及其对多聚谷氨酰胺疾病神经退行性变的影响，重点是聚集体相互作用蛋白的鉴定和调节聚集体形成的药物靶分子的鉴定。我们可以找到几个分子，它们调节聚集体的形成或降解。我们的方法对未来开发新疗法很重要。此外，我们可以开发一种新的基因疗法，可以特异性地降解异常的疾病蛋白。这种治疗在体内的效果本身揭示了聚集物在疾病过程中的病理意义。

We have been working about the aggregate formation and its effect on the neurodegeneration of polyglutamine diseases in this project, mainly focusing on the identification of the aggregate interacting proteins and of the target molecules for drugs modulating aggregate formation. We could find several molecules, which modulate aggregate formation or its degradation. Our approach is important for developing new therapies in future. Furthermore, we could develop a new gene therapy, in which abnormal disease protein could be specifically degraded. The effect of this treatment in vivo itself revealed the pathological significance of aggregates in the disease process.