The first human clinical trial of a gene therapy treatment for Parkinson’s disease is set to begin in the US, following successful results in animals.
The treatment almost completely abolished the jerky movements associated with the disorder in half of a group of mice, a team led by Matthew During of the University of Auckland showed.
The treatment involves using a harmless virus to shuttle a gene into a part of the brain called the subthalamic nucleus (STN). A loss of dopamine-producing cells in the brains of Parkinson’s patients causes over-activity in the STN, which in turn causes jerky movements. But when the cells take up the gene, their activity is suppressed.