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Cure found for Huntington disease in mice offers hope for treatment in humans

VANCOUVER, B.C.: Researchers at the University of British Columbia's Centre for Molecular Medicine and Therapeutics (CMMT) have provided ground-breaking evidence for a cure for Huntington disease in a mouse offering hope that this disease can be relieved in humans.

Published today in Cell journal, Dr. Michael Hayden and colleagues discovered that by preventing the cleavage of the mutant huntingtin protein responsible for Huntington disease (HD) in a mouse model, the degenerative symptoms underlying the illness do not appear and the mouse displays normal brain function. This is the first time that a cure for HD in mice has been successfully achieved.

"Ten years ago, we discovered that huntingtin is cleaved by 'molecular scissors' which led to the hypothesis that cleavage of huntingtin may play a key role in causing Huntington disease", said Dr. Michael Hayden, Director and Senior Scientist at the Child and Family Research Institute's Centre for Molecular Medicine and Therapeutics. Dr. Hayden is also a Canada Research Chair in Human Genetics and Molecular Medicine.

Now a decade later, this hypothesis has resulted in a landmark discovery. "This is a monumental effort that provides the most compelling evidence of this hypothesis to date", said Dr. Marian DiFiglia, Professor in Neurology, Massachusetts General Hospital, Harvard Medical School and one of the world's leading experts on Huntington disease. "Dr. Hayden and his team have shown in convincing fashion that many of the changes seen in HD patients can be erased in HD mice simply by engineering a mutation into the disease gene that prevents the protein from getting cleaved at a specific site".

To explore the role of cleavage, Dr. Hayden's team established an animal model of HD that replicated the key disease features seen in patients. A unique aspect of this particular animal model is that it embodied the human HD gene in exactly the same way seen in patients. This replication al
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Contact: Alexandra Howard
alexandra@cmmt.ubc.ca
604-875-3881
University of British Columbia
16-Jun-2006


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