Finding ways to improve treatment for hemophilia A by addressing protein issues
Overcoming FVIII protein misfolding and cell toxicity
This study is looking at a new way to help people with hemophilia A by using gene therapy to deliver a missing protein to their liver, with the hope of creating a safer and longer-lasting treatment.
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-11023049 on NIH RePORTER |
What this research studies
This research focuses on hemophilia A, a bleeding disorder caused by a deficiency of clotting factor VIII (FVIII). The team is exploring gene therapy using adeno-associated virus (AAV) to deliver FVIII to liver cells, aiming to overcome challenges such as the need for high doses and the decline in FVIII expression over time. They are investigating the misfolding of FVIII proteins that leads to cell toxicity and liver damage, with the goal of developing a more effective and long-lasting treatment for patients. By understanding the cellular responses to FVIII expression, the researchers hope to enhance the safety and efficacy of gene therapy for hemophilia A.
Who could benefit from this research
Good fit: Ideal candidates for this research are males diagnosed with hemophilia A who experience frequent bleeding episodes due to FVIII deficiency.
Not a fit: Patients with hemophilia A who do not respond to FVIII treatments or those with other bleeding disorders may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to a more effective and durable treatment for hemophilia A, reducing the frequency of bleeding episodes and improving patients' quality of life.
How similar studies have performed: Previous research has shown promise in using AAV-mediated gene therapy for hemophilia, but this specific approach to address FVIII misfolding is novel and untested.
Where this research is happening
Indianapolis, United States
- Indiana University Indianapolis — Indianapolis, United States (Active)
Researchers
- Principal investigator: Kaufman, Randal J. — Indiana University Indianapolis
- Study coordinator: Kaufman, Randal J.
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.