TREAT-HD: Delivering therapies to prevent neurodegeneration in Huntington's disease

Huntington's disease (HD) is caused by a faulty gene resulting in the build-up of a toxic protein. This damages brain cells, leading to problems with movement, thinking and behaviour. We know that the earliest signs of damage occur more than 20 years before the appearance of clinical symptoms. We believe this will be the optimum time to intervene with treatments. To find out if this is true we will: - Examine cell and mouse models to investigate how variability within the HD gene drives disease and whether drugs directed at these celluar processes have therapeutic benefit. - Study a group of young adult gene carriers decades before we expect symptoms to begin. Using brain imaging and assessments of behaviour we will obtain a detailed understanding of how the disease affects the brain over time. - Combine this information to develop measures which will help us determine whether future treatments are effective.