John D Heiss, Abhik Ray-Chaudhury, David E Kleiner, Debra J Ehrlich, Gretchen Scott, Nancy A Edwards, David S Goldstein, Dima A Hammoud, Piotr Hadaczek, Victor S Van Laar, Shantelle A Graff, Peter Herscovitch, Codrin Lungu, Mark Hallett, Russell R Lonser, Kareem A Zaghloul, Krystof S Bankiewicz
OBJECTIVE: Gene therapy by convection-enhanced delivery of type 2 adeno-associated virus-glial cell derived neurotrophic factor (AAV2-GDNF) to the bilateral putamina seeks to increase GDNF gene expression and treat Parkinson's disease (PD). METHODS: A 63-year-old man with advanced PD received AAV2-GDNF in a clinical trial. He died from pneumonia after anterior cervical discectomy and fusion 45 months later. An autopsy included brain examination for GDNF transgene expression...
May 8, 2024: Movement Disorders: Official Journal of the Movement Disorder Society