RNA molecules to control blood clotting
RNA Aptamers as Probes and Modulators of Coagulation
Researchers are designing small RNA-based molecules that stick to specific clotting proteins so blood clotting can be turned down or quickly reversed for people who need precise control of anticoagulation.
Quick facts
| Grant type | P01 program project |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Children's Hosp of Philadelphia NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project ID | NIH-11193795 on NIH RePORTER |
What this research studies
This project uses high-resolution X-ray structures to design RNA aptamers that bind specific blood clotting factors and then links those aptamers to small-molecule inhibitors to make very potent, targeted blockers. The team will test these bifunctional constructs in laboratory experiments and animal models to measure how well they stop clotting and how specifically they act. They will also develop short 'antidote' oligonucleotides that can neutralize the RNA portion and rapidly reverse the effect. Initial proof-of-concept work centers on thrombin before applying the approach to other clotting factors.
Who could benefit from this research
Good fit: People at high risk of dangerous blood clots or those who require anticoagulation that may need rapid reversal (for example around surgery or invasive procedures) are most likely to benefit from these advances.
Not a fit: Patients whose health problems do not involve abnormal clotting or who cannot receive nucleic acid–based treatments are unlikely to benefit directly from this research.
Why it matters
Potential benefit: If successful, this work could produce highly specific anticoagulants that are very powerful yet can be reversed quickly, improving safety for patients who need temporary or tightly controlled blood thinning.
How similar studies have performed: Related aptamer-plus-antidote approaches have shown feasibility in human testing, but the specific bifunctional EXosite-ACTive site (EXACT) design described here is a newer, more bioinspired strategy.
Where this research is happening
Philadelphia, United States
- Children's Hosp of Philadelphia — Philadelphia, United States (Active)
Researchers
- Principal investigator: Sullenger, Bruce Alan — Children's Hosp of Philadelphia
- Study coordinator: Sullenger, Bruce Alan
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.