AAV Gene Therapy Vectors: How they enter cells
Official title: Adeno-Associated Virus Gene Therapy Vectors: Molecular Interactions on Cell Entry
Funded at University of Missouri-Columbia · NIH-11393060 · Data updated 2026-11-12
A gene-delivery tool called AAV is being studied to understand exactly how it gets into human cells, with the goal of making gene therapies safer and more effective.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Missouri-Columbia NIH-funded |
| Lab location | 1 site (Columbia, United States) |
| Project start | August 1, 2017 |
| Project end | July 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $765,461 |
| NIH project number | 5R35GM122564-09 |
| Project ID | NIH-11393060 on NIH RePORTER |
Who could benefit from this research
✓Could help
Patients with genetic diseases who might benefit from future AAV-mediated gene therapies could be impacted by these discoveries.
✕Less likely to help
This basic science project does not directly recruit patients or test therapies in people.
What this research studies
This project looks at how AAV entry into human cells happens at the molecular level, focusing on receptors and the steps that allow the virus to move from the cell surface to the nucleus. Researchers will map interactions between AAV and proteins like AAVR and GPR108, and study how structural changes enable endosomal escape. They will use advanced imaging techniques to visualize AAV with receptor components and will connect these findings to the virus’s ability to deliver genetic material. The ultimate aim is to inform safer, lower-dose gene therapies for patients with genetic diseases.
Why it matters
Potential benefit: If successful, the work could lead to more efficient AAV-based therapies that require smaller doses and have fewer immune-related risks.
How similar studies have gone: Prior work has identified AAV receptors and endosomal escape mechanisms, with ongoing translational efforts in gene therapy.
Where this research is happening
-
University of Missouri-Columbia
Columbia, United States Active
Researchers
- Principal investigator: Chapman, Michael S. — University of Missouri-Columbia
- Study coordinator: Chapman, Michael S.
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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.