mRNA delivery tools to get medicines into body cells
Construction of in vivo mRNA delivery systems
This project builds new lipid-based carriers to carry mRNA into specific body cells, with the goal of enabling treatments like producing missing blood clotting protein for people with hemophilia.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| 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-11261608 on NIH RePORTER |
What this research studies
The team is designing and testing new lipid materials that can safely carry mRNA inside the body and release it into targeted cell types. They combine these lipids with mRNA and study how well the packages deliver messages, how long they last, and whether they are safe in animals. Early work showed the approach can produce human factor VIII in hemophilia A mice and enable other applications like base editing and macrophage-based infection control. If these delivery systems prove reliable and safe, they could be moved toward human testing at clinical sites.
Who could benefit from this research
Good fit: Patients with conditions that could be treated by mRNA-based approaches—such as hemophilia A or other genetic or infectious diseases targeted by mRNA—would be the likely candidates for future clinical trials of these delivery systems.
Not a fit: People seeking an immediate approved therapy or whose conditions cannot be addressed by mRNA delivery are unlikely to benefit directly from this preclinical research.
Why it matters
Potential benefit: If successful, the work could make mRNA treatments safer and more effective, potentially allowing single-dose or targeted therapies (for example, supplying missing clotting factor in hemophilia).
How similar studies have performed: Related lipid nanoparticle technologies have powered mRNA vaccines and shown promise in preclinical gene-replacement studies, but translating new delivery systems into safe, effective human treatments remains novel and ongoing.
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.