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Gladstone scientists identify protein form linked to Huntington's disease
Date:10/30/2011

SAN FRANCISCO, CAOctober 30, 2011Scientists at the Gladstone Institutes have discovered how a form of the protein linked to Huntington's disease influences the timing and severity of its symptoms, offering new avenues for treating not only this disease, but also a variety of similar conditions.

In a paper being published today in Nature Chemical Biology, the laboratory of Gladstone Senior Investigator Steven Finkbeiner, MD, PhD, singles out one form of a misfolded protein in neurons that best predicts whether the neuron will die. Neuronal death is key to the development of Huntington's symptomsincluding erratic behavior, memory loss and involuntary muscle movement. This research underscores the value of the cross-disciplinary work done at Gladstonea leading and independent biomedicalresearch organizationwhile revealing techniques that scientists anywhere can apply to conditions involving misfolded proteins, such as Alzheimer's disease and type 1 diabetes.

"Effective treatments for diseases such as Huntington's and Alzheimer's have been slow to develop," said Dr. Finkbeiner, whose research at Gladstone investigates the interactions between genes, neurons and memory. "We hope that our newfound understanding of precisely which misfolded proteins contribute to disease symptoms will speed up drug development for sufferers."

Huntington's, an ultimately fatal disease that affects more than a quarter of a million people nationwide, is caused by mutations in the gene that creates the huntingtin, or htt, protein. As the mutated gene produces htt, a segment of the protein called polyglutamine is mistakenly expanded, distorting htt's natural shape and function. As a result, the misfolded protein malfunctions and can be toxic.

Previous research into Huntington's found that misfolded proteins in the brain lead to the dysfunction and subsequent death of neurons. In this study, Dr. Finkbeiner set out to find which misfolded htt t
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Contact: Anne Holden
anne.holden@gladstone.ucsf.edu
415-734-2534
Gladstone Institutes
Source:Eurekalert  

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