Gene therapy for Parkinson's Disease - Discover Rush (Rush Univ Med Ctr Chicago)



Neurodevelopmental Disabilities

Parkinson's disease - Gene therapy

Gene therapy for Parkinson’s disease

Promising results from a phase one study of gene therapy could offer hope to patients with Parkinson’s disease. Although only 12 patients participated in the trial and results may have been biased by a placebo effect, the gene therapy tested at Rush University Medical Center reduced Parkinson’s disease symptoms by 40 percent. The phase one study was designed to determine how a drug is broken down and how it interacts with the human body to reveal some of the side effects associated with increasing doses. If these results are confirmed by future studies, gene therapy could offer the first chance to slow, halt or even reverse the course of Parkinson’s disease. Parkinson’s disease occurs when cells in the brain that produce dopamine, a chemical messenger needed for smooth and coordinated muscle movement, die or become impaired — resulting in shaking, stiffness, balance problems and other symptoms.

In the new therapy, neurosurgeons inject a gene into the brain that instructs the brain to produce a protein to protect and regenerate the dopamine-producing cells damaged by Parkinson’s disease.



Archive of Past Issues Discover Rush, 2007 - Spring Parkinson's disease - Gene therapy