Grant: Doctoral Award - Frederick Banting and Charles Best Canada Graduate Scholarships

Huntington Disease (HD) is an inherited disorder that results in uncontrolled movements in early stages and loss of movement in later stages of the disease. Patients also have cognitive deficits and psychiatric disorders. These symptoms are due to the malfunction and the gradual loss of neurons in various parts of the brain. There is currently no cure or treatment that alters the progression of HD. One of the reasons why neurons do not work properly and die, in HD, is that they contain suboptimal amounts of a molecule called GM1. GM1 helps neurons to communicate with each other and to respond to their environment. We have discovered that supplying GM1 to a mouse model of HD results in a dramatic improvement of the motor symptoms that are typical of this disease. My project involves understanding whether GM1 can stop disease progression and treat psychiatric and cognitive symptoms beside treating motor problems. A better understanding of the extent of GM1 effects in animal models of HD is crucial to bring our findings to the clinics and treat HD patients. In addition, my project is also focused on determining what portion of the GM1 molecule is necessary for its protective effects, so that this information can be used to make better drugs that can be given to HD patients by simple injections, rather than by drug infusion into the brain, as we do with GM1 in HD mice. My studies have direct clinical relevance and will potentially lead to the development of a therapy for HD.