Creating a new gene therapy to reduce seizures in epilepsy patients
Development and validation of a viral vector for targeted inhibition of DG granule cells
This study is testing a new gene therapy that could help people with drug-resistant epilepsy by targeting certain brain cells to reduce seizures, and it will first be tried out in mice before looking at how it might work for humans.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Emory University NIH-funded |
| Lab location | 1 site (Atlanta, United States) |
| Project ID | NIH-10877119 on NIH RePORTER |
What this research studies
This research focuses on developing a novel gene therapy aimed at treating drug-resistant epilepsy by targeting specific brain cells known as dentate gyrus granule cells. The approach involves using a viral vector to deliver a gene that can inhibit these cells, potentially reducing the frequency of seizures. The researchers will first test the effectiveness of this gene therapy in a mouse model before considering its application in humans. By understanding how seizures are initiated and propagated, this research aims to provide new treatment options for patients who do not respond to existing medications.
Who could benefit from this research
Good fit: Ideal candidates for this research are adults over 21 years old who suffer from drug-resistant epilepsy.
Not a fit: Patients with epilepsy that is well-controlled by existing medications may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to a groundbreaking treatment for patients with drug-resistant epilepsy, significantly improving their quality of life.
How similar studies have performed: While gene therapy for epilepsy is a relatively novel approach, preliminary studies have shown promise in targeting specific brain cells to manage seizures.
Where this research is happening
Atlanta, United States
- Emory University — Atlanta, United States (Active)
Researchers
- Principal investigator: Gutekunst, Claire-Anne N — Emory University
- Study coordinator: Gutekunst, Claire-Anne N
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.