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Fox Foundation grant funds major gene therapy advance for treatment of Parkinson's disease

An innovative gene therapy approach pioneered by Pennsylvania-based RheoGene Inc. will be further refined and tested in preliminary clinical trials within four years, thanks to a $4.2 million grant from the Michael J. Fox Foundation for Parkinson's Research (MJFF).

A wholly owned affiliate of the University of Pittsburgh Medical Center (UPMC), RheoGene Inc. has developed technology to manage gene expression, a key component of gene-based therapies. RheoGene's therapeutic system uses a patented small-molecule mediator that can turn genes "on" or "off" as well as adjust the level of gene activity similar to the way a rheostat regulates electric current.

"This project has the potential to revolutionize the clinical application of gene therapy not only for the millions of people with Parkinson's disease, but for countless numbers afflicted by other health ailments as well," said Deborah W. Brooks, MJFF president and CEO. "It is a natural fit with the foundation's commitment to drive innovative technology that will have a significant impact on patients' lives."

Parkinson's disease gradually destroys brain cells that produce dopamine, a chemical messenger crucial for the cellular communication that controls muscle movement. As dopamine levels drop, symptoms increase. These include tremors in the arms, legs and face; periodically stiff or frozen limbs; slow movement; and impaired balance and coordination. In the United States, at least 500,000 and perhaps as many as 1.5 million people are thought to have Parkinson's disease, with 50,000 new cases being diagnosed each year, according to the National Institute of Neurological Disorders and Stroke, a division of the National Institutes of Health. Because risk increases with age, these rates will increase as the population ages.

Current treatment options focus on replacing lost dopamine with the drug levodopa (L-dopa), which converts to dopamine in the brain, or brain surgery to control tremo
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