Via EurekAlert!: “A gene therapy called NLX-P101 dramatically reduces movement impairment in Parkinson’s patients, according to results of a Phase 2 study … The approach introduces a gene into the brain to normalize chemical signaling. … The study is the first successful randomized, double-blind clinical trial of a gene therapy for Parkinson’s or any neurologic disorder … Half of patients receiving gene therapy achieved dramatic symptom improvements, compared with just 14 percent in the control group. Overall, patients receiving gene therapy had a 23.1 percent improvement in motor score, compared to a 12.7 percent improvement in the control group. … Improved motor control was seen at one month and continued virtually unchanged throughout the six-month study period. … Gene therapy is the use of a gene to change the function of cells or organs to improve or prevent disease. To transfer genes into cells, an inert virus is used to deliver the gene into a target cell. In this case, the glutamic acid decarboxylase (GAD) gene was used because GAD makes a chemical called GABA, a major inhibitory neurotransmitter in the brain that helps ‘quiet’ excessive neuronal firing related to Parkinson’s disease. … In Parkinson’s disease, not only do patients lose many dopamine-producing brain cells, but they also develop substantial reductions in the activity and amount of GABA in their brains. This causes a dysfunction in brain circuitry responsible for coordinating movement.”

Link: http://www.eurekalert.org/pub_releases/2011-03/nyph-gtr031411.php