Drug Discovery

Cognitive Enhancement

Distal cis-elements and compounds that regulate tau splicing

Investigator(s): Jianhua Zhou, Ph.D.

Institution(s): University of Massachusetts Medical School

Duration:

Summary:

Alzheimer's disease has two major pathological characteristics, amyloid plaques and aggregates of tau protein (tangles). Tau aggregation is also the major feature of at least 20 other neurodegenerative disorders, classified as tauopathies that include frontotemporal dementia and Parkinsonism linked to chromosome 17 (FTDP-17). More than 29 mutations in the tau gene have been identified from FTD patients. A group of these mutations alter the processing of tau protein causing tau accumulation. In this project, the investigator will establish a faithful cell based assay for identification of drugs that prevent tau accumulation. The assay will then be used for high throughput screening in collaboration with the Harvard Laboratory for Drug Discovery in Neurodegeneration which has an extensive and diverse compound collection and the capacity to facilitate the rapid follow up of lead candidates with its substantial drug development capabilities.