Lung-targeted mRNA therapy for Bourneville-related LAM using specialized lipid nanoparticles
Official title: Develop lung-targeted synthetic lipid nanoparticles for mRNA medicine treating pulmonary lymphangioleiomyomatosis
Funded at Tufts University Medford · NIH-11298940 · Data updated 2026-11-12
A new lung-focused mRNA therapy using tiny lipid particles aims to slow or stop lung damage in people with lymphangioleiomyomatosis who have mutations in TSC1 or TSC2.
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 | 2026 |
| Award (this year) | $632,655 |
| NIH project number | 5R01HL171728-03 |
| Project ID | NIH-11298940 on NIH RePORTER |
Who could benefit from this research
✓Could help
Patients with lymphangioleiomyomatosis due to TSC1/TSC2 mutations who have progressive lung disease and are candidates for advanced therapies.
✕Less likely to help
Patients without LAM or those without TSC1/TSC2 mutations may not benefit from this targeted approach.
What this research studies
This grant supports developing lung-targeted lipid nanoparticles that deliver functional mRNA to TSC-deficient lung cells to suppress disease pathways. The approach builds on preliminary data showing targeted delivery and tumor reduction in models of LAM, with the goal of a safer, more effective treatment that could complement or extend beyond current therapies. Researchers will optimize the delivery system to reach lung tissue specifically and test its effect on mTORC1 signaling and tumor burden in preclinical models. If successful, this could move toward clinical testing to help patients with LAM who experience progressive lung decline.
Why it matters
Potential benefit: If successful, patients with LAM could receive a more durable treatment that targets the underlying genetic cause and potentially reduces the need for lung transplantation.
How similar studies have gone: Previous work shows that mRNA delivery to lung tissue can reduce tumor burden in LAM models, with some promising preclinical results guiding this project.
Where this research is happening
-
Tufts University Medford
Boston, United States Active
Researchers
- Principal investigator: Xu, Qiaobing — Tufts University Medford
- Study coordinator: Xu, Qiaobing
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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.