AAV gene therapy to improve healing of large bone defects
A novel, clinically expedient, AAV-based gene therapy for bone healing
This work is testing a gene therapy that uses a viral vector to help adults with large bone gaps heal stronger and faster.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Mayo Clinic Rochester NIH-funded |
| Lab location | 1 site (Rochester, United States) |
| Project ID | NIH-11269156 on NIH RePORTER |
What this research studies
Researchers are developing a gene therapy that delivers the bone-building protein BMP-2 using an adeno-associated virus (AAV) to improve healing of large bone defects. They will study how inflammation, especially the molecule interleukin‑1 (IL‑1), changes the way BMP‑2 works and the quality of new bone. The team will use a validated rat femur model with both male and female animals to measure bone formation, strength, and quality. The goal is to create an effective, affordable approach that could move toward testing in people with critical-size bone injuries.
Who could benefit from this research
Good fit: Adults with large segmental bone defects, nonunions, or bone loss from trauma or tumor surgery would be the most likely candidates for this kind of therapy in the future.
Not a fit: People with simple, small fractures, children, or patients with active bone infection or certain immune conditions would likely not benefit from this specific therapy.
Why it matters
Potential benefit: If successful, this approach could offer a safer, more affordable way to heal large or hard-to-heal bone gaps and reduce the need for repeat surgeries or large bone grafts.
How similar studies have performed: Previous animal studies showed viral delivery of BMP‑2 can heal large bone gaps but adenoviral approaches were limited by immune reactions, and AAV-based delivery is a newer strategy with promising preclinical support but not yet proven in humans.
Where this research is happening
Rochester, United States
- Mayo Clinic Rochester — Rochester, United States (Active)
Researchers
- Principal investigator: Evans, Christopher Howard — Mayo Clinic Rochester
- Study coordinator: Evans, Christopher Howard
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.