Gene therapy to calm overactive movement-control brain cells in Parkinson’s
Gene therapy targeting striatal dysfunction for Parkinson’s disease
A gene therapy that lowers specific glutamate receptors in the movement-control area of the brain to help people with Parkinson's who have troublesome motor symptoms.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Emory University NIH-funded |
| Lab location | 1 site (Atlanta, United States) |
| Project ID | NIH-11259459 on NIH RePORTER |
What this research studies
This work aims to develop a gene therapy that reduces certain NMDA glutamate receptor subunits in striatal neurons linked to abnormal movement in Parkinson's. Researchers are advancing from encouraging rodent results into detailed non-human primate studies to check safety and effects on motor signs. The approach uses viral delivery to knock down GluN2 subunits in the part of the brain that becomes overactive when dopamine is lost. If these preclinical steps go well, the therapy could move toward testing in people with Parkinson's.
Who could benefit from this research
Good fit: People with Parkinson's disease who have persistent or disabling motor symptoms despite standard medications would be the most likely candidates for future trials.
Not a fit: People whose Parkinson's problems are mainly non-motor, or who cannot tolerate brain-directed gene therapies for medical reasons, are unlikely to benefit from this approach.
Why it matters
Potential benefit: If successful, the therapy could lower pathological striatal activity and improve motor symptoms and responses to dopamine replacement in Parkinson's disease.
How similar studies have performed: Preclinical work, including rodent gene knockdown and selective NMDA receptor blockade, has shown promising effects on motor symptoms, but human testing has not yet occurred.
Where this research is happening
Atlanta, United States
- Emory University — Atlanta, United States (Active)
Researchers
- Principal investigator: Papa, Stella M — Emory University
- Study coordinator: Papa, Stella M
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.