Restoring defective protein homeostasis in frontotemporal dementia

额颞性痴呆(FTD)与有毒蛋白在病变脑区的病理性堆积和聚集有关。该项目将利用一种新型的高通量药物筛选平台技术、FTD患者来源的神经细胞和遗传小鼠模型来筛选药物，以提高有毒蛋白质的清除和神经细胞的健康。这种方法应该会加速发现潜在的治疗FTD根本原因的药物，以努力减缓疾病的进展。

Frontotemporal dementia (FTD) is associated with pathological accumulation and aggregation of toxic proteins in affected brain regions. This project will employ a novel high throughput drug screening platform technology, FTD patient-derived nerve cells and genetic mouse models to screen drugs to improve clearance of toxic proteins and nerve cell health. This approach should accelerate discovery of agents to potentially treat the underlying cause of FTD in an effort to slow disease progression.