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Archive - Jul 21, 2013

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Scientists ID Potential Target to Reduce Deadly Protein Buildup in Huntington’s Disease

Alzheimer's, Huntington's, Parkinson's--these are names forever linked to what they represent: diseases that ravage the brain's neurons and leave entire regions to wither and die. These and other so-called neurodegenerative diseases are often associated with the buildup of toxic proteins that lead to the death of neurons. But now, scientists at the Gladstone Institutes have discovered that the progression of disease is not due to the buildup of toxins itself, but rather in the individual neurons' ability to flush these toxins out. Further, they have identified a therapeutic target that could boost this ability, thereby protecting the brain from the diseases' deadly effects. In an article published online on July 21, 2013 in Nature Chemical Biology, researchers in the laboratory of Gladstone Investigator Steve Finkbeiner, M.D., Ph.D., describe how a newly developed technology allowed them to see—for the first time—how individual neurons fight back against the buildup of toxic proteins over time. Focusing their efforts on a model of Huntington's disease, the team observed how different types of neurons in the brain each responded to this toxic buildup with different degrees of success, offering clues as to why the disease causes neurons in one region to die, while neurons in another are spared. "Huntington's—an inherited and fatal disorder that leads to problems with muscle coordination, cognition, and personality—is characterized by the toxic buildup of a mutant form of the huntingtin protein in the brain," explained Dr. Finkbeiner, who directs the Taube-Koret Center for Neurodegenerative Disease Research at Gladstone. Dr. Finkbeiner is also a professor of neurology and physiology at the University of California, San Francisco, with which Gladstone is affiliated.