Targeted treatment for genetic brain disorders causing seizures
Molecular profiling of seizures to provide comprehensive, durable, and targeted treatment for inherited white matter disorders
['FUNDING_OTHER'] · RESEARCH INST NATIONWIDE CHILDREN'S HOSP · NIH-11001789
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
| Phase | ['FUNDING_OTHER'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | RESEARCH INST NATIONWIDE CHILDREN'S HOSP (nih funded) |
| Locations | 1 site (COLUMBUS, UNITED STATES) |
| Trial ID | NIH-11001789 on ClinicalTrials.gov |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research are children diagnosed with leukodystrophies, particularly those with Vanishing White Matter Disease or Alexander's Disease.
Not a fit: Patients with other forms of neurological disorders not related to leukodystrophies may not benefit from this research.
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 performed: 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
COLUMBUS, UNITED STATES
- 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
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.
Conditions: Alexander Disease, Alexander syndrome, Aran-Duchenne disease