Rochester Researchers Receive $700,000 for Gene Therapy Research

October 30, 1996

University of Rochester scientists have received a three-year, $700,000 grant from the National Institutes of Health to develop new ways to modify genes to help treat neurodegenerative diseases such as Parkinson's disease, amyotrophic lateral sclerosis (Lou Gehrig's disease), and Alzheimer's disease.

Martha Bohn, associate professor in the Department of Neurobiology and Anatomy, and Howard Federoff, professor in the Department of Neurology, will use the funds to search for ways to shuttle vital proteins into the brain. In patients with Parkinson's disease, for example, nerve cells that produce the neurotransmitter dopamine gradually die, causing tremors, rigidity, and difficulty in walking. Scientists have discovered proteins that help keep these nerve cells alive, but getting the substances into the brain is a challenge. Federoff and Bohn are working on ways to genetically alter brain cells so that they'll be able to produce the proteins continuously, eliminating the need for ongoing drugs or injections.

"Using gene therapy to spur the brain to produce vital substances offers intriguing possibilities to treat a variety of neurological disorders," Bohn says. "In our experiments we'll investigate ways to deliver therapeutic genes specifically to the cells that need them, to lessen both the symptoms of disease and the side effects of treatment."

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