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DNA nanoparticles hold promise in gene therapy for Parkinson's disease

University of Kentucky researcher David Yurek was recently awarded $66,000 by The Michael J. Fox Foundation for Parkinson's Research (MJFF) under the foundation's Rapid Response Innovation Awards program. The goal of this newly launched initiative is to move quickly to support innovative research focused on the cause of and cure for Parkinson's disease (PD). In particular, MJFF seeks to fund high-risk, high-reward projects tackling critical scientific roadblocks that if successful, can open new avenues for PD therapy development.

Yurek's project, titled "Nanoparticle Gene Therapy for Parkinson's Disease," examines a relatively new gene therapy approach for treating neurodegenerative disorders. He is testing the feasibility of using a novel technology to condense DNA plasmids into nanoparticles and deliver them to the brain as a means to halt or prevent the neurodegenerative process.

The technology comes from Copernicus Therapeutics, Inc., a biotechnology company in Cleveland, Ohio. Yurek, whose laboratory is one of the first to apply this technology to central nervous system disorders, said this relatively new gene therapy strategy holds potential to help repair faulty genes. It entails transduction, a technique for expressing a particular gene in a cell by delivering DNA into the cell and making the cell synthesize the protein that corresponds to that DNA.

"We plan to use this technology to transduce brain cells so that they express proteins beneficial to the cell's survival," Yurek said.

The MJFF award will allow Yurek to test the feasibility of delivering condensed DNA nanoparticles that encode for a neurotrophic factor to the brain as a means to halt or prevent the neurodegenerative process in an animal model of PD. Neurotrophic factors are capable of protecting neurons from dying, thereby rescuing essential neurons in the brain. In animal studies, neurotrophic factors have revived dormant brain cells, caused them to
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Contact: Hollye Staley
Hollye.Staley@uky.edu
859-323-6363
University of Kentucky
1-Mar-2007


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