{"id":"25e635d9-8fe4-4023-b40d-299d18d6b3c4","slug":"from-the-bench-to-the-bedside-gene-therapy-for-parkinsons-disease-the-roles-of-the-habenula-and-nucleus-accumbens-in-depression-microglia-and-neurodegeneration","title":"From the bench to the bedside: Gene therapy for Parkinson’s disease, The roles of the habenula and nucleus accumbens in depression, Microglia and neurodegeneration","authors":["Ausaf A. Bari","Jason S. Hauptman"],"abstract":"Background: Gene therapy for Parkinson's disease aims to address the disinhibition of the subthalamic nuclei (STN), which contributes to the symptoms of the disease. The study investigates the efficacy of AAV2-GAD gene therapy, which delivers the GAD gene to the STN to enhance GABA production and potentially reduce motor symptoms. Methods: In this double-blind, sham-surgery controlled, randomized trial, patients with advanced Parkinson's disease were assigned to receive either bilateral injections of AAV2-GAD into the STN or a sham procedure. Patients were followed for up to 6 months post-surgery to assess changes in motor symptoms using the UPDRS motor score. Results: Patients treated with AAV2-GAD exhibited a significant improvement of approximately 23% in motor symptoms compared to sham controls at 6 months. Additionally, there were reports of reduced freezing episodes and improved medication effects, with the therapy demonstrating a favorable safety profile. Conclusion: This study represents a landmark advancement in gene therapy for neurological diseases, showing that AAV2-GAD is both safe and potentially effective in treating Parkinson's disease. The findings support further exploration of gene therapy as a viable treatment option in neurosurgery. Keywords: gene therapy, Parkinson's disease, AAV2-GAD, subthalamic nucleus, GABA, motor symptoms, safety, clinical trial","thumbnailUrl":null,"publishDate":"2011-06-09T00:00:00.000Z","doi":"10.4103/2152-7806.81967","categories":["Original Article"],"fullTextUrl":"https://surgicalneurologyint.com/api/articles/25e635d9-8fe4-4023-b40d-299d18d6b3c4/pdf"}