Gene Therapy of Parkinson’s Disease

The gene for GAD, the enzyme responsible for biosynthesis of GABA, was delivered to cells in the subthalamic nucleus of patients with Parkinson’s disease by means of a genetically modified virus. Functional MRI scans show (a) decreased activation of the subthalamic nucleus and (b) increased activation of the supplementary motor area. (c) Relationship between changes in the activation of the supplementary motor area and symptoms of Parkinson’s disease. (From Kaplitt, M. G., Feigin, A., Tang, C., et al. Lancet, 2007, 369, 2097–2105. Reprinted with permission.)