Blocking antibodies to make AAV gene therapy work for more people
Novel strategy to block Nabs for AAV gene delivery
Testing a protein that temporarily blocks antibodies so more people can receive AAV-based gene treatments safely.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Univ of North Carolina Chapel Hill NIH-funded |
| Lab location | 1 site (Chapel Hill, United States) |
| Project ID | NIH-11301001 on NIH RePORTER |
What this research studies
This project is developing a protein called Protein-M that binds antibodies and can hide the neutralizing antibodies that prevent AAV gene vectors from working. Researchers will test Protein-M in lab experiments and animal models to see whether it protects AAV vectors without changing which tissues the virus targets. They will compare different doses and AAV types, measure how much vector activity is restored, and monitor for immune or other side effects. If lab and animal results are favorable, the team aims to advance toward approaches that could allow people with prior AAV exposure to receive gene therapies.
Who could benefit from this research
Good fit: People who need or are candidates for AAV-based gene therapy but have pre-existing neutralizing antibodies against AAV.
Not a fit: People who do not need AAV gene therapy, who lack anti-AAV antibodies, or who cannot receive protein-based immunomodulation are unlikely to benefit.
Why it matters
Potential benefit: Could allow people who already have antibodies to AAV to receive AAV gene therapies they were previously excluded from.
How similar studies have performed: Other strategies to bypass AAV antibodies (capsid changes, masking, plasmapheresis, antibody-cleaving enzymes) have had limited success or side effects, and the Protein-M protein approach is newer with promising early laboratory results.
Where this research is happening
Chapel Hill, United States
- Univ of North Carolina Chapel Hill — Chapel Hill, United States (Active)
Researchers
- Principal investigator: Li, Chengwen — Univ of North Carolina Chapel Hill
- Study coordinator: Li, Chengwen
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.