Nanoparticles to carry gene-based medicines into the brain
Development of novel lipid nanoparticles for nucleic acids delivery in the brain
This project is creating biodegradable lipid nanoparticles to carry RNA medicines across the blood–brain barrier to reach brain cells affected by Alzheimer's disease.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Icahn School of Medicine at Mount Sinai NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project ID | NIH-11314605 on NIH RePORTER |
What this research studies
You should know researchers are designing special lipid nanoparticles that protect RNA and help it cross the blood–brain barrier to reach neurons. They are testing different nanoparticle formulations and using animal models to see which versions deliver mRNA into the brain while avoiding the liver. The team is also engineering the RNA itself and targeting cell surface transporters to improve delivery to specific brain cell types linked to Alzheimer's. If these preclinical steps work, the approach could move toward human trials of brain-targeted nucleic acid therapies.
Who could benefit from this research
Good fit: People with Alzheimer’s disease would be the eventual candidates for clinical trials that use these brain-targeted RNA therapies.
Not a fit: Because this is early, preclinical work, people seeking immediate treatment or those without Alzheimer’s should not expect direct benefit now.
Why it matters
Potential benefit: If successful, this could enable safe, effective delivery of gene-based treatments to brain cells and potentially slow or reverse Alzheimer's-related brain changes.
How similar studies have performed: Lipid nanoparticles have been highly successful for mRNA vaccines and show promise in animal brain-delivery studies, but reliable, safe delivery to human brains remains largely unproven.
Where this research is happening
New York, United States
- Icahn School of Medicine at Mount Sinai — New York, United States (Active)
Researchers
- Principal investigator: Dong, Yizhou — Icahn School of Medicine at Mount Sinai
- Study coordinator: Dong, Yizhou
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.