Grant: Project Grant

To date, the problem in Huntington's Disease (HD) has been thought to result from a genetic mutation which leads to the production of an abnormal protein - called mutant huntingtin - within brain cells. Over time, this protein causes the cells to slowly stop working and die. In recent years, new evidence has emerged which suggests that other factors may also contribute to the death of these nerve cells but these factors were thought to be of only minor significance for the disease. However, we found that this may not be the case as we detected the abnormal mutant huntingtin protein in healthy transplanted tissue implanted in the brains of affected individuals. This indicates that mutant huntingtin had spread and entered the implanted healthy graft tissue. These findings have been repeated in both cell and animal models and it is now accepted that mutant huntingtin can be transported between cells. We now seek to explore how mutant huntingtin spread relates to disease presentation and how prevention or reduction of this spread by vaccines could diminish disease burden. If true, the theory that we propose would both improve our understanding of what goes wrong in HD and, more importantly, potentially lead to new treatment options.