Improving gene editing in neurons to treat Rett syndrome
Official title: Establishing and Optimizing a Prime Editing Method in Neurons for Treatment of Rett Syndrome
Funded at Univ of Massachusetts Med Sch Worcester · NIH-11004385 · Data updated 2026-11-12
This study is exploring a new way to fix the genetic changes that cause Rett syndrome in brain cells, using a special technique that could lead to better treatments for this condition affecting infants.
Quick facts
| Grant type | Fellowship grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Univ of Massachusetts Med Sch Worcester NIH-funded |
| Lab location | 1 site (Worcester, United States) |
| Project start | January 15, 2023 |
| Project end | February 3, 2025 |
| Funding year | 2025 |
| Award (this year) | $7,006 |
| NIH project number | 5F31NS127555-03 |
| Project ID | NIH-11004385 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are infants diagnosed with Rett syndrome who have mutations in the MECP2 gene.
✕Less likely to help
Patients with Rett syndrome who do not have MECP2 mutations or those with advanced stages of the disease may not benefit from this research.
What this research studies
This research focuses on developing a new method for gene editing in neurons to correct mutations responsible for Rett syndrome, a condition that affects infants and leads to severe developmental issues. The approach utilizes prime editing, a cutting-edge technology that allows for precise modifications of genes. By delivering the necessary components via lipid nanoparticles, the researchers aim to enhance the efficiency of gene editing specifically in brain cells. This could potentially lead to more effective treatments that address the root cause of the disease rather than just managing symptoms.
Why it matters
Potential benefit: If successful, this research could significantly improve the quality of life for patients with Rett syndrome by correcting the underlying genetic mutations.
How similar studies have gone: While prime editing is a relatively novel approach, previous studies using CRISPR technologies have shown promise in gene editing, suggesting potential for success in this area.
Where this research is happening
-
Univ of Massachusetts Med Sch Worcester
Worcester, United States Active
Researchers
- Principal investigator: Keener, David G. — Univ of Massachusetts Med Sch Worcester
- Study coordinator: Keener, David G.
Related research
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Neuronal Cholesterol Enzyme CYP46A1 and Rett Syndrome biology
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-
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A study to find measurable brain signals that show whether treatments are helping children with Rett syndrome.
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MeCP2 reactivation on the inactive X to treat Rett syndrome
This project explores turning on the silent MECP2 gene on the inactive X chromosome to help people with Rett syndrome feel better.
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Studying how deep brain stimulation helps Rett syndrome in mice
Researchers are testing how deep brain stimulation affects brain cells and memory in a mouse model of Rett syndrome to uncover mechanisms that could guide future therapies.
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Molecular Pathways Behind Rett Syndrome in Humans
A patient-focused look at how changes in the MECP2 gene affect brain function and development in Rett syndrome.
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.