Safer gene therapy options for Hemophilia A
Toward Safer Gene Therapy for Hemophilia A
Developing liver-directed gene therapy that is safer and keeps factor VIII working longer for people with Hemophilia A.
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-11325354 on NIH RePORTER |
What this research studies
If you have Hemophilia A, this project aims to find out why liver-directed gene therapy can sometimes cause liver injury and why factor VIII levels fall over time. Researchers will examine how AAV gene vectors and the factor VIII gene interact with liver cells and the immune system, using laboratory experiments and analysis of samples from clinical trials. They will test changes to the gene constructs and delivery methods to improve secretion of factor VIII and reduce stress on the liver. The overall aim is to produce approaches that maintain factor VIII levels longer while reducing liver side effects.
Who could benefit from this research
Good fit: Adult males with Hemophilia A (severe factor VIII deficiency) who are potential candidates for liver-directed AAV gene therapy.
Not a fit: People with significant pre-existing liver disease, high levels of neutralizing antibodies to AAV, children, or those ineligible for liver-directed approaches may not benefit from these specific efforts.
Why it matters
Potential benefit: Could lead to gene therapies that provide longer-lasting factor VIII correction with fewer liver-related side effects.
How similar studies have performed: Earlier AAV liver gene therapy trials for Hemophilia A showed promising early correction but have reported declining factor VIII over time and some liver toxicity, so this work builds on those mixed results.
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.