Huntington's Disease
Huntington's Disease Research Articles

The opportunity to find novel and useful therapies for Huntington's disease is currently greater than ever. We would like, as the core of these studies, to do specific pre-clinical experiments to test new ideas for gene therapy or replacement for the cells most vulnerable to the Huntington gene in the striatum. In this way, we would create a preventive strategy for protecting or replacing cells prior to the development of Huntington's disease, as well as the potential for repair once such cells have started to succumb to the toxicity of the gene causing dysfunction or death of the striatal neuron. We are also evaluating human cases that have received fetal cells to understand how, similar stem cell or neurogenesis could re-establish connectivity to normalize brain structure and function.

In addition, we are actively exploring the mechanisms that make some neurons, in particular, in the caudate putamen region of HD patients so vulnerable to the mutation in huntingtin. We have discovered many intracellular gateways that are compromised by the HD mutation. Current work is aimed at restoring intracellular enzymes and coping mechanisms in vulnerable cell systems. In addition, we are working on gene-expression blockades in the most vulnerable neurons.