Investigating gene therapy for hemophilia A using AAV vectors
Biology of Subgenomic AAV Vector Particles
This study is looking at a new gene therapy for people with hemophilia A, a bleeding disorder, by using a special virus to help deliver the right gene and make treatment safer and more effective.
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-11023060 on NIH RePORTER |
What this research studies
This research focuses on developing a gene therapy approach for hemophilia A, a bleeding disorder caused by defects in the F8 gene. The team is exploring the use of adeno-associated virus (AAV) vectors to deliver the necessary gene therapy, aiming to overcome challenges such as decreasing levels of the factor VIII protein and potential safety concerns. By analyzing the complexity of AAV vector populations using advanced sequencing techniques, the researchers hope to improve the effectiveness and safety of gene therapy for patients with hemophilia A.
Who could benefit from this research
Good fit: Ideal candidates for this research are males diagnosed with hemophilia A who may benefit from innovative gene therapy approaches.
Not a fit: Patients with other types of hemophilia or bleeding disorders unrelated to the F8 gene may not receive benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to a curative gene therapy for hemophilia A, significantly improving the quality of life for affected patients.
How similar studies have performed: Previous research using AAV vectors for gene therapy has shown promising results, although this specific approach is still being refined and tested.
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.