Gene therapy to induce immune tolerance in dogs with hemophilia A
Immune tolerance induction by AAV-FVIII gene therapy for canine hemophilia A with inhibitors
This study is testing a new gene therapy for dogs with hemophilia A who have developed issues with their treatment, aiming to help their bodies accept a key protein they need for blood clotting, which could make their care easier and less costly.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Children's Hosp of Philadelphia NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project ID | NIH-10851822 on NIH RePORTER |
What this research studies
This research investigates a new gene therapy approach to treat hemophilia A in dogs, specifically targeting those that have developed inhibitors to factor VIII. The therapy uses adeno-associated virus (AAV) vectors to deliver the canine F8 gene, which is responsible for producing factor VIII, directly to the liver. By doing so, the aim is to induce immune tolerance, allowing the dog's body to accept factor VIII without producing harmful antibodies. This could potentially reduce the need for expensive and prolonged treatment regimens currently in use.
Who could benefit from this research
Good fit: Ideal candidates for this research are dogs diagnosed with hemophilia A who have developed inhibitors to factor VIII.
Not a fit: Dogs without hemophilia A or those who do not have inhibitors to factor VIII may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could provide a more effective and less costly treatment option for dogs suffering from hemophilia A with inhibitors.
How similar studies have performed: Previous studies using AAV gene therapy for hemophilia A without inhibitors have shown promising results, indicating potential for success in this novel approach.
Where this research is happening
Philadelphia, United States
- Children's Hosp of Philadelphia — Philadelphia, United States (Active)
Researchers
- Principal investigator: Samelson-Jones, Ben J — Children's Hosp of Philadelphia
- Study coordinator: Samelson-Jones, Ben 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.