Using a new method to edit genes to treat Rett Syndrome
Official title: Application of a novel dCasNano-based Epigenetic Editing to Treat Rett Syndrome
Funded at Epitor Therapeutics, INC. · NIH-11008487 · Data updated 2026-11-12
This study is exploring a new way to use gene editing to help girls with Rett Syndrome by trying to turn back on a gene that isn’t working properly, and they’ll be testing this method on brain cells grown from Rett patients to see if it’s safe and effective.
Quick facts
| Grant type | Sbir 1 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Epitor Therapeutics, INC. NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project start | September 15, 2024 |
| Project end | February 28, 2026 |
| Funding year | 2024 |
| Award (this year) | $698,459 |
| NIH project number | 1R43NS139830-01 |
| Project ID | NIH-11008487 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are girls diagnosed with Rett Syndrome, particularly those with mutations in the MeCP2 gene.
✕Less likely to help
Patients with Rett Syndrome who do not have mutations in the MeCP2 gene may not benefit from this research.
What this research studies
This research focuses on developing a novel gene editing technique to treat Rett Syndrome, a severe neurodevelopmental disorder primarily affecting girls. The approach utilizes a specialized CRISPR system to reactivate a silenced gene responsible for the condition, aiming to restore normal function in affected neurons. By packaging this gene editing tool into a virus, researchers hope to deliver it directly to the brain, where it can effectively target and modify the genes involved in Rett Syndrome. The study will involve testing this method in lab-grown neurons derived from Rett patients to ensure its safety and effectiveness.
Why it matters
Potential benefit: If successful, this research could lead to a groundbreaking treatment that restores normal gene function in Rett Syndrome patients, potentially improving their neurological outcomes.
How similar studies have gone: While gene editing for neurodevelopmental disorders is a relatively novel approach, preliminary studies have shown promise in similar methodologies, indicating potential for success.
Where this research is happening
-
Epitor Therapeutics, INC.
New York, United States Active
Researchers
- Principal investigator: Tsai, Yi-Ting — Epitor Therapeutics, INC.
- Study coordinator: Tsai, Yi-Ting
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MeCP2 reactivation on the inactive X to treat Rett syndrome
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Studying how deep brain stimulation helps Rett syndrome in mice
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Molecular Pathways Behind Rett Syndrome in Humans
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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.