Understanding how adeno-associated viruses assemble for gene therapy
Mechanistic Studies of AAP and Capsid Assembly of AAV Vectors
This study is looking at how certain viruses used in gene therapy are made, especially focusing on a special protein that helps form their protective shell, with the goal of making these therapies better for patients.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Oregon Health & Science University NIH-funded |
| Lab location | 1 site (Portland, United States) |
| Project ID | NIH-10754553 on NIH RePORTER |
What this research studies
This research investigates the assembly process of adeno-associated viruses (AAV), which are used as vectors in gene therapy. The focus is on understanding how these viruses are produced, particularly the role of a protein called assembly-activating protein (AAP) in forming the virus's protective shell. By studying the mechanisms of AAV capsid assembly, the research aims to improve the efficiency and quality of AAV vector production, which is crucial for effective gene therapy. Patients may benefit from advancements in gene therapy that arise from these findings.
Who could benefit from this research
Good fit: Ideal candidates for this research are patients with genetic disorders that could be treated using gene therapy involving adeno-associated viruses.
Not a fit: Patients with conditions that are not amenable to gene therapy or those who do not have genetic disorders may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to more effective and cost-efficient gene therapies for various genetic conditions.
How similar studies have performed: Previous research has shown promise in understanding AAV assembly, but this specific approach focusing on AAP and serotype differences is relatively novel.
Where this research is happening
Portland, United States
- Oregon Health & Science University — Portland, United States (Active)
Researchers
- Principal investigator: Nakai, Hiroyuki — Oregon Health & Science University
- Study coordinator: Nakai, Hiroyuki
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.