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Huntington's disease

Huntington's disease is a fatal hereditary disorder. The children of a Huntington's patient have a 50/50 chance of inheriting the disease. While 30,000 people in the United States currently suffer from the disease, over 150,000 individuals are at risk of developing Huntington's. 

Huntington's results from a mutation in the gene coding for a protein known as "huntingtin." If a person has this genetic mutation, he or she will develop the disease. Without the mutation, a person will not develop Huntington's nor pass it on to children.

The normal huntingtin protein is present in all brain cells and is required for cell survival. However, when the gene is mutated, the resulting protein is altered and behaves differently, making some brain cells vulnerable.  For unknown reasons the mutant protein only affects certain parts of the brain.  Affected areas include the cortex (which controls planning, thought, and memory) and the basal ganglia (which coordinates movement).

There is no treatment nor cure for Huntington's disease. Standard antidepressants, tranquilizers, and antipsychotic drugs may help temper the emotional impact of the disease.

Examples of Harvard NeuroDiscovery Center initiatives to fight Huntington's include: 

  • Understanding the disease.  Now that the huntingtin gene and protein have been pinpointed, scientists at the Harvard NeuroDiscovery Center are identifying the mechanisms that cause certain brain cells to self-destruct.

  • Discovering drugs.  We collaborate with investigators across the neuroscience academic community to transform scientific discoveries into new drug discovery opportunities. Today, we are working on several new "compounds" that may prove worthy for further development as a new treatment for Huntington's.

  • Clinical trials.  Studies by Harvard NeuroDiscovery Center scientists suggest that creatine, fatty acids, and vitamin C have positive effects in animal models of Huntington's disease. The Harvard NeuroDiscovery Center is pursuing these encouraging results by examining what effects these substances have in human patients.

The Harvard NeuroDiscovery Center is dedicated to accelerating the discovery of effective treatments and cures for Huntington's disease.  For information about our many important initiatives and how to support our approach, click on "Advance The Cause" at left. For information about coping with Huntington's, the following websites might be of interest:  Huntington's Disease Society of America