Grant: Fellowship - Priority Announcement: Huntington's Disease

Huntington's Disease (HD) is a genetic neurodegenerative disorder that affects families worldwide. HD is caused by a mutation in a single gene encoding the huntingtin protein. Affected individuals have a 50% chance of passing this gene on to each child. The symptoms of the disease include involuntary movement, loss of motor coordination, cognitive impairment and psychiatric disturbances, caused by degeneration of brain cells and leading to death, ten to twenty years after onset. Currently, no treatment or cure exists for HD. Studies in the Truant research group focus on the normal function of the huntingtin protein, in hopes of finding novel drug targets for HD. One of the discoveries recently made in our laboratory is that the huntingtin protein is involved in a cell stress response. We found that this stress response is altered in cells with mutant huntingtin. Cell stress is an important factor in many neurological diseases, in fact, a similar stress response is dysfunctional in Alzheimer's disease. We have developed biological tools that can be used to compare the stress response in normal cells versus mutant huntingtin cells in response to a range of potential drugs. The goal is to identify potential drugs that cure the stress response in mutant huntingtin cells and to pursue these compounds to be further developed in collaboration with pharmaceutical companies such as CHDI Inc. The proposed experiments will advance our understanding of normal and mutant huntingtin function, and provide leads for the therapeutic intervention of HD. These advances may have implications in other neurodegenerative diseases such as Alzheimer's Disease.