Gene editing therapies to prevent epileptic seizures
Official title: Gene editing therapeutics that prevent epileptic seizures
Funded at University of Texas Hlth Science Center · NIH-11605225 · Data updated 2026-11-12
A new gene-editing approach aims to reduce seizures in people with epilepsy by directly modifying a brain gene tied to the condition.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Texas Hlth Science Center NIH-funded |
| Lab location | 1 site (San Antonio, United States) |
| Project start | September 9, 2026 |
| Project end | August 31, 2027 |
| Funding year | 2026 |
| Award (this year) | $565,436 |
| NIH project number | 1R56NS146956-01A1 |
| Project ID | NIH-11605225 on NIH RePORTER |
Who could benefit from this research
✓Could help
Individuals with epilepsy caused by or linked to SCN8A-related mechanisms who may be candidates for future gene-editing therapies.
✕Less likely to help
Patients whose seizures are due to other genetic factors or non-genetic causes may not benefit from this approach.
What this research studies
This project explores using CRISPR-based gene editing to target the SCN8A gene, which is linked to epileptic seizures. By delivering a precise edit to stop the harmful allele in neurons, the goal is to reduce seizure activity while avoiding broad disruption of healthy brain function. The research builds on previous animal studies and seeks a deliverable closer to clinical use, addressing challenges like delivery to the brain and immune responses. If successful, this work could move toward new, more targeted therapies for patients with difficult-to-control epilepsy.
Why it matters
Potential benefit: If successful, patients with epilepsy could experience fewer seizures and improved quality of life through a targeted genetic treatment.
How similar studies have gone: Previous studies have shown that allele-specific gene editing can reduce seizures in animal models, but clinical translation remains from early-stage to human applications.
Where this research is happening
-
University of Texas Hlth Science Center
San Antonio, United States Active
Researchers
- Principal investigator: Lee, Hye Young — University of Texas Hlth Science Center
- Study coordinator: Lee, Hye Young
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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.