Targeted gene silencing in the brain using siRNA helpers
Official title: Design and implementation of siRNA conjugates as tools for CNS cell-specific gene modulation
Funded at Vanderbilt University · NIH-11605743 · Data updated 2026-11-12
This grant tests a new way to turn off specific brain genes in particular CNS cell types using siRNA conjugates to help understand brain biology and disease.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Vanderbilt University NIH-funded |
| Lab location | 1 site (Nashville, UNITED STATES) |
| Project start | September 3, 2026 |
| Project end | September 2, 2027 |
| Funding year | 2026 |
| Award (this year) | $708,389 |
| NIH project number | 1R56EB040350-01 |
| Project ID | NIH-11605743 on NIH RePORTER |
Who could benefit from this research
✓Could help
Patients with CNS disorders could benefit in the long term if these CNS-targeted gene-silencing tools translate to therapies.
✕Less likely to help
Healthy individuals or patients without CNS involvement may not benefit from this basic science tool development.
What this research studies
Researchers from Vanderbilt University are developing siRNA conjugates that can deliver gene silencing directly to defined brain cells, aiming to overcome limitations of traditional CNS gene targeting methods. By tuning the siRNA, delivery route, and dose, they want to achieve precise, cell-specific activity after a single administration route into the brain. The project builds on prior lipid-siRNA conjugates that spread through the mouse CNS after injection, seeking to improve targeting while reducing off-target effects. If successful, this work could provide a more flexible tool for studying brain function and disease mechanisms in humans.
Why it matters
Potential benefit: A more precise way to study and potentially treat brain diseases by silencing harmful genes in specific brain cells.
How similar studies have gone: Earlier siRNA conjugates showed promising CNS distribution and activity, suggesting this approach could be effective with optimization.
Where this research is happening
-
Vanderbilt University
Nashville, United States Active
Researchers
- Principal investigator: Lippmann, Ethan — Vanderbilt University
- Study coordinator: Lippmann, Ethan
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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.