Creating targeted nanoparticles to deliver mRNA medicine for lung disease treatment
Official title: Develop lung-targeted synthetic lipid nanoparticles for mRNA medicine treating pulmonary lymphangioleiomyomatosis
Funded at Tufts University Medford · NIH-11074550 · Data updated 2026-11-12
This study is testing a new way to deliver special particles that carry messenger RNA directly to the lungs to help treat pulmonary lymphangioleiomyomatosis (LAM), a genetic lung condition, by aiming to restore a missing protein and improve lung health.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Tufts University Medford NIH-funded |
| Lab location | 1 site (Boston, UNITED STATES) |
| Project start | April 15, 2024 |
| Project end | February 29, 2028 |
| Funding year | 2025 |
| Award (this year) | $581,010 |
| NIH project number | 5R01HL171728-02 |
| Project ID | NIH-11074550 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with pulmonary lymphangioleiomyomatosis who have mutations in the TSC1 or TSC2 genes.
✕Less likely to help
Patients with LAM who do not have TSC1 or TSC2 mutations may not benefit from this research.
What this research studies
This research focuses on developing synthetic lipid nanoparticles that can specifically deliver messenger RNA (mRNA) to the lungs to treat pulmonary lymphangioleiomyomatosis (LAM), a genetic disorder that leads to severe lung damage. The approach involves designing nanoparticles that can effectively target and enter TSC2-deficient cells, which are crucial in the progression of LAM. By using these nanoparticles to deliver functional mRNA, the research aims to restore the missing protein and potentially reverse the disease's effects. The study will evaluate the effectiveness of this mRNA replacement therapy in preclinical models to assess its safety and efficacy.
Why it matters
Potential benefit: If successful, this research could provide a novel treatment option that may halt or reverse lung damage in patients with pulmonary lymphangioleiomyomatosis.
How similar studies have gone: Previous research has shown promising results with mRNA therapies in other genetic disorders, indicating potential success for this novel approach in LAM.
Where this research is happening
-
Tufts University Medford
Boston, United States Active
Researchers
- Principal investigator: Xu, Qiaobing — Tufts University Medford
- Study coordinator: Xu, Qiaobing
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.