A Clinical Study of the Safety and Efficacy of Chemogenetics Therapy in the Treatment of Parkinson's Disease
Summary
The investigators propose a gene therapy strategy using chemical genetic inhibition to intervene in the abnormal activity of the subthalamic nucleus in Parkinson's disease. The investigators design and construct a highly efficient therapeutic injection STP-001 (first drug), through the efficient adeno-associated virus capsid (AAV), neuronal promoter (hSyn), and chemical genetic effector element (hM4Di), and accurately inject the drug into the bilateral subthalamic nucleus, the core pathological nucleus of Parkinson's disease, through stereotactic technology. Combined with a very low dose of clozapine (the second drug), the abnormal activity of the subthalamic nucleus is precisely intervened to improve the core motor symptoms of Parkinson's disease.
Timeline
- Start
- 2025-08-01
- Primary completion
- 2025-12-30
- Completion
- 2025-12-30
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | STP-001 | Gene therapy (AAV / viral vector) | 1e+12 vg | Other |
| Subject | STP-001 | Gene therapy (AAV / viral vector) | 2e+12 vg | Other |
| Subject | STP-001 | Gene therapy (AAV / viral vector) | 4e+12 vg | Other |
| Background | Clozapine | Other / unclassified | 3.125 mg | Oral |