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 Director: Prof. Ole Isacson, Biography and Research
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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.

Our current neuroprotective gene therapy work is aimed at improving synaptic function, intracellular transport, and protein management in vulnerable neurons in Huntington's disease. These mechanisms are known to be dysfunctional in neurons in Huntington’s disease, and we propose that improving their function could slow or prevent neurodegenerative changes. In particular we are working on two synaptic proteins: Rab3B and Rab1A and components of the protein-degradation system. We are testing therapeutic delivery of these proteins using gene therapy approaches in animal models of Huntington’s disease.

 

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