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Gene therapy for Parkinson's disease moves forward in animals

afety net. Once we turn it on, it's on for life. So we have to control it, and we're working on this as we speak. But it's not ready for clinical trials."

About a half million Americans struggle with Parkinson's disease, including former Attorney General Janet Reno, former heavyweight boxing champion Muhammad Ali and film star Michael J. Fox, according to the National Institute for Neurological Disorders and Stroke. Pope John Paul II was recently hospitalized because of breathing problems that were complicated by his advancing Parkinson's disease.

"The use of GDNF as an approach against Parkinson's disease has truly had some ups and downs," said J. William Langston, M.D., scientific director and chief executive officer of The Parkinson's Institute in Sunnyvale, Calif., who recently chaired a panel probing GDNF experimentation for the Michael J. Fox Foundation for Parkinson's Research. "This is additional experimental evidence that suggests that it can be a promising approach to this disease using in vivo gene therapy, which is very applicable to humans. It even presents theoretical reasons that might solve some of the safety issues that have been raised about GDNF. But many things remain that we still don't understand."

The recent findings in laboratory animals were a joint effort of Lund University in Lund, Sweden, the University of Cambridge in the United Kingdom, and the McKnight Brain Institute and the Genetics Institute of the University of Florida. Scientists included internationally renowned Parkinson's expert Anders Björklund of Lund, a pioneer of the experimental treatment involving the transplantation of fetal cells into the brains of Parkinson's patients, and his colleague Deniz Kirik, a neurobiologist.

"This work with GDNF in combination with other regenerative medicine approaches, including stem cells, promises to have a place for both protection and repair in Parkinson's disease," said Dennis Steindler, Ph.D., director o
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Source:University of Florida


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