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Small molecules might block mutant protein production in Huntington's disease
Date:5/3/2009

be blocked to treat the disease effectively, he said. "Fifty percent inhibition might be enough, but that remains to be determined," Dr. Corey said.

In future studies, the researchers plan to try these RNA mimics in whole animals, using several different mutations of the genes.

Laurie Tompkins, who oversees neurogenetics grants at the National Institutes of Health's National Institute of General Medical Sciences, said the ability to control individual genes makes this work stand out.

"By exploiting processes that occur in normal cells, Dr. Corey has come up with a clever way to do this that may well lead to new ways to combat Huntington's and other related diseases," she said.


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Contact: Aline McKenzie
aline.mckenzie@utsouthwestern.edu
214-648-3404
UT Southwestern Medical Center
Source:Eurekalert

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