Targeted treatment for genetic brain disorders causing seizures
Official title: Molecular profiling of seizures to provide comprehensive, durable, and targeted treatment for inherited white matter disorders
Funded at Research Inst Nationwide Children's Hosp · NIH-11001789 · Data updated 2026-11-12
This study is exploring a new gene therapy to help kids with leukodystrophies, like Vanishing White Matter Disease and Alexander's Disease, by using a special virus to fix the genetic issues causing their symptoms, with hopes of improving their movement, weight, and overall health.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Research Inst Nationwide Children's Hosp NIH-funded |
| Lab location | 1 site (Columbus, United States) |
| Project start | July 15, 2024 |
| Project end | June 30, 2026 |
| Funding year | 2024 |
| Award (this year) | $38,359 |
| NIH project number | 1F99NS139538-01 |
| Project ID | NIH-11001789 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are children diagnosed with leukodystrophies, particularly those with Vanishing White Matter Disease or Alexander's Disease.
✕Less likely to help
Patients with other forms of neurological disorders not related to leukodystrophies may not benefit from this research.
What this research studies
This research focuses on genetic neurodegenerative diseases known as leukodystrophies, which affect the white matter of the brain and often present in childhood. The study aims to develop a targeted gene therapy using adeno-associated viruses to treat conditions like Vanishing White Matter Disease and Alexander's Disease, which currently have no effective treatments. By using a mouse model, researchers are investigating how this therapy can improve motor function, body weight, and extend survival while addressing severe seizures that arise from these conditions. Patients may benefit from a novel approach that targets the underlying genetic causes of their disorders.
Why it matters
Potential benefit: If successful, this research could provide a groundbreaking treatment option for children suffering from severe genetic brain disorders.
How similar studies have gone: Previous research has shown promise in using gene therapy for similar genetic conditions, indicating potential for success in this approach.
Where this research is happening
-
Research Inst Nationwide Children's Hosp
Columbus, United States Active
Researchers
- Principal investigator: Herstine, Jessica Audreyanna — Research Inst Nationwide Children's Hosp
- Study coordinator: Herstine, Jessica Audreyanna
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A study at Case Western Reserve University is exploring whether gently stimulating a natural brain pathway called the corpus callosum can help identify where seizures start and stop them from spreading in people with hard-to-control epilepsy.
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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.