Persistent GDNF Expression 45 Months after Putaminal Infusion of AAV2-GDNF in a Patient with Parkinson's Disease.

ABSTRACT:

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. Putaminal catecholamine concentrations were compared to in vivo ¹⁸F-Fluorodopa (¹⁸F-FDOPA) positron emission tomography (PET) scanning results before and 18 months after AAV2-GDNF infusion.

Results

Parkinsonian progression stabilized clinically. Postmortem neuropathology confir

SUBMITTER: Heiss JD 

PROVIDER: S-EPMC11341257 | biostudies-literature | 2024 Aug

REPOSITORIES: biostudies-literature