Gene therapy aims to treat a disease by introducing a new gene into a cell to replace or re-programme a function that has become defective.
In Parkinson’s this therapy is still largely experimental. The GAD gene is introduced which makes a small molecule called GABA. When introduced into the subthalamic nucleus (STN), GABA ‘re-sets’ and dampens the over-activity of brain cells which cause motor symptoms such as tremor and dyskinesia. It is non-invasive as the cells are introduced by inserting the GAD gene into a modified virus, and so far this therapy does not seem to produce the side effects associated with surgery.
Although much more research and clinical trials are needed, preliminary findings are promising1.
