Restoring defective protein homeostasis in frontotemporal dementia

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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.