Gene therapy for Parkinson's shows promise

NEWS IN BRIEF — Posted July 9, 2007

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The results of a phase I study of gene therapy for Parkinson's disease published in the Lancet last month indicate that this treatment strategy is safe and could improve symptoms.

Researchers injected glutamic acid decarboxylase genes carried by adeno-associated viruses into one hemisphere of the brains of a dozen patients. No adverse events were associated with the procedure, and symptoms on the side of the body controlled by the treated side of the brain improved in three months. The effect continued for at least a year, and PET scans detected changes in the area of the brain associated with this disease.

"These exciting results need to be validated in a larger trial, but we believe this is a milestone -- not only for the treatment of Parkinson's disease -- but for the use of gene-based therapies against neurological conditions generally," said Michael Kaplitt, MD, PhD, lead author and director of movement disorders surgery at New York-Presbyterian Hospital/Weill Cornell Medical Center.

Note: This item originally appeared at

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