16. First Gene Therapy For Parkinson’s Disease

Michael G. Kaplitt, Weill Cornell Medical College, Neurological Surgery, performed the world’s first gene therapy for Parkinson’s disease, marking the first-ever in vivo gene therapy in the brain for an adult neurological disease. The five-hour procedure, performed with the patient awake, was part of a FDA-approved phase I clinical trial, and the culmination of 15 years of research. The goal of Kaplitt’s gene therapy approach is to reset a specific group of cells that have become overactive in an affected part of the brain, causing the impaired movements of Parkinson’s disease. Kaplitt pinpoints the optimal location in the patient’s brain using information from an advanced 3T MRI image. This is merged with a CT scan, using the latest computer imaging technology. The final target is confirmed using fine electrical probes that identify the signature pattern of electrical activity of individual cells within the brain. The gene therapy agent (adeno-associated virus) is slowly delivered through a very fine catheter. After the infusion, the catheter is removed and the skin closed. This first of its kind clinical trial moves toward safe treatment of Parkinson’s disease and other brain disorders, using gene therapy.

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