A New Way to Deliver Factor VIII for Hemophilia A
Platelet-derived FVIII Gene Therapy of Hemophilia A
This research explores a new gene therapy approach for hemophilia A that uses platelets to deliver Factor VIII, especially for patients who develop antibodies against current treatments.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Medical College of Wisconsin NIH-funded |
| Lab location | 1 site (Milwaukee, United States) |
| Project ID | NIH-11128549 on NIH RePORTER |
What this research studies
For people with hemophilia A, especially those who develop antibodies to standard Factor VIII treatments, this research offers a new hope. We are working on a gene therapy that teaches your body's platelets to make Factor VIII right where it's needed. This method aims to overcome the problem of your immune system attacking the Factor VIII, potentially helping your body accept the new protein. The goal is to provide a long-lasting and effective way to manage hemophilia A.
Who could benefit from this research
Good fit: Ideal candidates for future applications of this research would be individuals with hemophilia A, especially those who have developed inhibitory antibodies against Factor VIII.
Not a fit: Patients without hemophilia A or those who do not develop inhibitory antibodies may not directly benefit from this specific gene therapy approach.
Why it matters
Potential benefit: If successful, this approach could offer a safe and effective long-term treatment for hemophilia A, particularly for patients who currently struggle with inhibitory antibodies.
How similar studies have performed: Previous studies in animal models have shown promising results, demonstrating sustained Factor VIII expression and immune tolerance using this platelet-targeted gene delivery method.
Where this research is happening
Milwaukee, United States
- Medical College of Wisconsin — Milwaukee, United States (Active)
Researchers
- Principal investigator: Shi, Qizhen — Medical College of Wisconsin
- Study coordinator: Shi, Qizhen
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.