Improving gene therapy for hemophilia A
Toward Safer Gene Therapy for Hemophilia A
This study is looking at ways to make gene therapy for hemophilia A safer and more effective by using a special virus to deliver the missing gene to the liver, so that patients can have a better 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-11023034 on NIH RePORTER |
What this research studies
This research focuses on enhancing the safety and effectiveness of gene therapy for hemophilia A, a bleeding disorder caused by a deficiency in factor VIII. The team is investigating the use of adeno-associated viral (AAV) gene transfer to deliver the necessary gene to the liver, which has shown promise in initial trials but faces challenges such as declining factor levels and liver toxicity. By addressing these safety concerns and understanding the interactions between the gene therapy vector and liver cells, the research aims to provide a more durable and safer treatment option for patients. The approach includes studying the immune response and potential risks associated with the therapy.
Who could benefit from this research
Good fit: Ideal candidates for this research are males diagnosed with severe hemophilia A who are experiencing challenges with current treatment options.
Not a fit: Patients with mild hemophilia or those who do not have hemophilia A may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to a safer and more effective gene therapy that provides lasting treatment for hemophilia A.
How similar studies have performed: Previous clinical trials using similar gene therapy approaches have shown initial success, but this research aims to address critical safety and efficacy concerns that have emerged.
Where this research is happening
Indianapolis, United States
- Indiana University Indianapolis — Indianapolis, United States (Active)
Researchers
- Principal investigator: Herzog, Roland W. — Indiana University Indianapolis
- Study coordinator: Herzog, Roland W.
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.