How the outer shell of AAV gene therapy viruses interacts with cells and the immune system
AAV capsids and their cellular interactions
Researchers are looking at how the outer shell (capsid) of AAV gene therapy viruses binds to human cells and antibodies to help make gene therapies safer and work for more people.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Florida NIH-funded |
| Lab location | 1 site (Gainesville, United States) |
| Project ID | NIH-11261243 on NIH RePORTER |
What this research studies
From your perspective as a patient, the team is examining how the protein coat of AAV gene therapy vectors attaches to cell surfaces and moves inside cells. They will use blood samples and antibodies from donors and clinical trial participants to see where and how those antibodies stick to the capsid. The project combines structural methods (to map binding sites on the capsid) with functional tests (to see how those interactions block delivery or cause immune reactions). Findings will guide changes to capsids to avoid neutralizing antibodies and improve delivery while lowering harmful immune responses.
Who could benefit from this research
Good fit: People who are candidates for AAV-based gene therapies or who have prior exposure to AAV and can provide blood samples would be the most directly relevant participants.
Not a fit: Patients whose conditions are not treated by AAV gene therapies or who will never be candidates for gene therapy are unlikely to receive direct benefit from this project.
Why it matters
Potential benefit: If successful, this work could allow more people to receive AAV gene therapies by reducing pre-existing antibody blocks and lowering dose-related toxicities.
How similar studies have performed: Several AAV gene therapies have already been approved and shown clinical benefit, but immune reactions to capsids remain an ongoing challenge that this work aims to address.
Where this research is happening
Gainesville, United States
- University of Florida — Gainesville, United States (Active)
Researchers
- Principal investigator: Mckenna, Robert — University of Florida
- Study coordinator: Mckenna, Robert
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.