Developing new systems to deliver mRNA to specific cells in the body
Construction of in vivo mRNA delivery systems
This study is working on new ways to deliver messenger RNA (mRNA) directly to the right cells in the body using tiny fat particles, which could help improve treatments for conditions like hemophilia and other genetic disorders.
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-11005744 on NIH RePORTER |
What this research studies
This research focuses on creating innovative delivery systems for messenger RNA (mRNA) that can target specific cell types in the body. The approach involves developing lipid-based nanoparticles that can safely and effectively deliver mRNA, which is crucial for treating various conditions, including hemophilia. The researchers will test these systems in animal models to evaluate their efficiency, safety, and ability to produce therapeutic proteins. By enhancing the delivery of mRNA, this research aims to improve treatment options for patients with specific genetic disorders.
Who could benefit from this research
Good fit: Ideal candidates for this research are patients with genetic disorders, such as hemophilia A, who may benefit from improved mRNA therapies.
Not a fit: Patients with conditions that do not involve mRNA therapies or those who are not responsive to genetic treatments may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to more effective treatments for genetic disorders by improving the delivery of therapeutic mRNA to targeted cells.
How similar studies have performed: Previous research has shown promise in using lipid nanoparticles for mRNA delivery, indicating that this approach has potential for success.
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.