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Gene Therapy Stops Parkinson's Mutation

Posted on: Wednesday, 18 January 2006, 21:00 CST

Northwestern University scientists say they're developing a therapy to selectively turn off a gene involved in Parkinson's disease development.

The therapy was designed by Martha Bohn and colleagues at Northwestern University's Feinberg School of Medicine. Bohn is director of the neurobiology program at Northwestern's Children's Memorial Research Center and professor of pediatrics, molecular pharmacology and biological chemistry.

The technique removes a protein known as alpha-synuclein from the diseased dopamine-producing neurons that die in Parkinson's disease. Alpha-synuclein is abundant in structures known as Lewy bodies -- a diagnostic hallmark of Parkinson's disease.

This is the first step in developing a new therapy for Parkinson's disease based on molecular knowledge of the disease, said Mohan Sapru, research assistant professor of pediatrics, first author of the study and co-inventor of the gene therapy technology. It may also be useful for other diseases of the brain, such as dementia with Lewy bodies, a disease also characterized by Lewy bodies in the brain.

The research is described in the January online issue of the journal Experimental Neurology.


Source: United Press International

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