Understanding how the presence of DNA affects the structure of AAV capsids
Official title: Structural and chemical changes between empty and full AAV capsids
Funded at Michigan Technological University · NIH-10833669 · Data updated 2026-11-12
This study is looking at the differences between empty and full virus shells used in gene therapy to help make treatments safer and more effective for patients.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Michigan Technological University NIH-funded |
| Lab location | 1 site (Houghton, United States) |
| Project start | May 1, 2023 |
| Project end | April 30, 2026 |
| Funding year | 2024 |
| Award (this year) | $186,408 |
| NIH project number | 5R21AI175913-02 |
| Project ID | NIH-10833669 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals who may benefit from gene therapy for genetic disorders or diseases.
✕Less likely to help
Patients who are not candidates for gene therapy or those with conditions unrelated to AAV treatments may not benefit from this research.
What this research studies
This research investigates the structural and chemical differences between empty and full adeno-associated virus (AAV) capsids, which are important for gene therapy. By using advanced techniques like atomic force microscopy and mass spectrometry, the study aims to uncover how the presence of the viral genome influences the capsid's structure and surface properties. The goal is to improve the manufacturing process of AAVs to reduce the number of empty capsids, which can cause adverse effects in patients. This could lead to safer and more effective gene therapy products.
Why it matters
Potential benefit: If successful, this research could lead to safer gene therapy treatments with fewer side effects for patients.
How similar studies have gone: Previous research has shown promise in improving gene therapy vectors, but this specific approach to understanding AAV capsid structure is relatively novel.
Where this research is happening
-
Michigan Technological University
Houghton, United States Active
Researchers
- Principal investigator: Heldt, Caryn — Michigan Technological University
- Study coordinator: Heldt, Caryn
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.