Study on the mechanism of aggregate formation in neurodegeneration

点击翻译按钮获取中文摘要

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.