Developing RNA-based treatments for blood clotting and bleeding disorders
Official title: RNA-based therapeutics for hemostasis and thrombosis
Funded at Thomas Jefferson University · NIH-11081013 · Data updated 2026-11-12
This study is exploring new RNA-based treatments to help people with blood and vascular diseases, especially those at risk of blood clots or bleeding, by safely targeting specific genes in blood cells to improve their care.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Thomas Jefferson University NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project start | May 1, 2024 |
| Project end | April 30, 2026 |
| Funding year | 2025 |
| Award (this year) | $491,398 |
| NIH project number | 5R33HL173910-02 |
| Project ID | NIH-11081013 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals with thrombotic disorders or bleeding disorders who have not responded well to existing treatments.
✕Less likely to help
Patients with non-blood-related conditions or those who do not have issues with blood clotting or bleeding may not benefit from this research.
What this research studies
This research focuses on creating innovative RNA-based gene therapies aimed at improving the management of blood and vascular diseases, particularly those related to thrombotic risks and bleeding disorders. The approach involves using noncoding RNA to selectively suppress genes in platelets and megakaryocytes, which are crucial for blood clotting. By administering these therapies through intravenous or subdermal methods, the goal is to achieve targeted effects without the side effects commonly associated with current treatments. This could lead to more effective and safer management options for patients suffering from these conditions.
Why it matters
Potential benefit: If successful, this research could provide patients with more effective and safer treatments for managing blood clotting and bleeding disorders.
How similar studies have gone: While RNA-based therapies are a growing field, this specific approach of using noncoding RNA for targeted gene suppression in blood cells is relatively novel and has not been extensively tested in clinical settings.
Where this research is happening
-
Thomas Jefferson University
Philadelphia, United States Active
Researchers
- Principal investigator: Goldfinger, Lawrence E — Thomas Jefferson University
- Study coordinator: Goldfinger, Lawrence E
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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.