Brain gene editing with safe lipid nanoparticles
Official title: mRNA/Lipid nanoparticle-mediated genome editing in the brain.
Funded at Upstate Medical University · NIH-11442324 · Data updated 2026-11-12
A new approach uses tiny fat-based particles to deliver gene editing tools to the brain to help treat neurological diseases.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Upstate Medical University NIH-funded |
| Lab location | 1 site (Syracuse, United States) |
| Project start | August 15, 2026 |
| Project end | July 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $244,500 |
| NIH project number | 1R21EB040117-01 |
| Project ID | NIH-11442324 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are individuals with genetic or acquired brain disorders that could be addressed by brain-wide or region-specific gene editing in future trials.
✕Less likely to help
This preclinical work does not involve enrolling patients or testing a therapy in people at this stage.
What this research studies
This project aims to develop a safer, more precise way to deliver gene editing components to brain cells using sulfonium-based lipid nanoparticles. Researchers will study how these particles enter brain tissue and how they carry mRNA and CRISPR/Cas9 cargo to target cells. They will test the editing system in preclinical brain models to see if it can achieve meaningful genome changes with controlled dosing. If successful, this work could lay the groundwork for future therapies for inherited and acquired brain disorders.
Why it matters
Potential benefit: If successful, patients with certain brain disorders could benefit from new gene-editing therapies delivered directly to the brain.
How similar studies have gone: Prior work shows that similar lipid nanoparticle approaches can deliver genetic cargo to brain cells, with ongoing efforts to improve efficiency and safety.
Where this research is happening
-
Upstate Medical University
Syracuse, United States Active
Researchers
- Principal investigator: Men, Yuqin — Upstate Medical University
- Study coordinator: Men, Yuqin
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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.