Letter from the Scientists

Our research mission is to understand how the presence of the Huntington’s Disease (HD) mutation affects cells in the brain and to determine ways to slow or prevent the onset and progression of the disease.  To this end, our goal is to identify ways to treat HD.  At the forefront of these are strategies to restore the ability of HD neurons to prevent the accumulation of the toxic protein that causes HD, to make appropriate levels of important brain proteins, to restore the activity of the normal Huntingtin protein, to harness the power of stem cells for transplantation into the brain, and to use stem cells derived from skin samples from HD patients to generate the “disease in a dish” to better understand HD and test drugs. For each of these strategies, there are experimental results in HD model systems that demonstrate their promise, such as in cells and mice that take on disease “symptoms.” And excitingly –we can now test drugs in HD mice that “target” cell processes that the group has studied over the years.  Testing in HD mice and in human stem cells may provide key information about whether a given drug or approach may ultimately benefit patients. There is considerable hope on the horizon to treat this devastating disease.
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Leslie Thompson, PhD
​Joan Steffan, PhD
Larry Marsh, PhD