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Chemical chaperone could open door to treatment of neurological disorder
Date:2/5/2008

the amount of mutant NPC1 that folds into a functional form.

"The screening can be done in as little as two weeks because the facility in Rockville, Md., has a huge library of compounds and state-of-the-art robotic equipment that can perform the tests at very high speed," Ory explains. "Then we will bring the compounds that show a positive effect to our laboratory and validate them on cell lines from Niemann-Pick patients. After that we will work with a pharmaceutical partner to take the ones that are effective in cells and make sure they will be safe and effective in people."

Although Niemann-Pick disorders are rare, affecting fewer than 2,000 people worldwide, Ory says that it is likely the chemical chaperone approach to therapy could also be useful for other disorders caused by genetic mutations that lead to protein misfolding. This includes cystic fibrosis, a lung and digestive system disorder that affects 70,000 children and adults around the world.


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Contact: Gwen Ericson
ericsong@wustl.edu
314-286-0141
Washington University School of Medicine
Source:Eurekalert  

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