The biological and pathological functions of TDP-43

神经退行性疾病如MND的社会和经济负担是巨大的。这种疾病和许多其他相关疾病的关键组织病理学标志是蛋白质TDP-43的沉积。我们的研究旨在了解其在很大程度上未知的功能，例如通过产生转基因动物模型。这些将成为开发TDP-43靶向药物治疗的基础。这项研究的国家利益是多方面的：通过破译基本的生物机制，为新数据申请专利，为无法治愈和致命的疾病制定治疗策略，并扩大与澳大利亚生物技术和国际制药公司的联系。

The social and economic burden of neurodegenerative such as MND is enormous. A key histopathological hallmark of this and many other related diseases are deposits of the protein TDP-43. Our research aims at understanding its largely unknown functions, for example by generating transgenic animal models. These will form the base for the development for a TDP-43-directed drug treatment. The national benefit of this research is manifold: by deciphering basic biological mechanisms, patenting new data, developing treatment strategies for un-curable and fatal disorders, and expanding links to Australian biotech and international pharmaceutical companies.