How partial AAV gene therapy particles behave
Biology of Subgenomic AAV Vector Particles
This project looks at how incomplete AAV gene therapy particles form and whether they affect gene therapy for people with hemophilia A.
Quick facts
| Grant type | P01 program project |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Indiana University Indianapolis NIH-funded |
| Lab location | 1 site (Indianapolis, United States) |
| Project ID | NIH-11325361 on NIH RePORTER |
What this research studies
Researchers will use advanced DNA sequencing to examine the mix of AAV particles made for hemophilia A gene therapy and identify incomplete or 'subgenomic' forms. They will focus on a particular snapback genome that might produce double-stranded RNA and change how much factor VIII stays in the blood. The team will use laboratory experiments and animal models to trace whether these particles contribute to falling factor VIII levels or abnormal liver cell growth. The aim is to understand the causes of these problems so AAV gene therapy can be safer and more reliable for patients.
Who could benefit from this research
Good fit: Ideal participants would be people with hemophilia A who have had, are considering, or can provide blood or tissue samples related to AAV-FVIII gene therapy.
Not a fit: People without hemophilia A or those who are ineligible for AAV-based treatments (for example, due to high anti-AAV antibodies) are unlikely to benefit directly from this project.
Why it matters
Potential benefit: If successful, this work could make AAV-based gene therapy for hemophilia A safer and more likely to give lasting improvement in clotting factor levels.
How similar studies have performed: Previous AAV gene therapy trials for hemophilia A have shown promise but have also raised questions about long-term durability and safety, so this work builds on prior progress while addressing remaining risks.
Where this research is happening
Indianapolis, United States
- Indiana University Indianapolis — Indianapolis, United States (Active)
Researchers
- Principal investigator: Xiao, Weidong — Indiana University Indianapolis
- Study coordinator: Xiao, Weidong
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.