Improving gene therapy for hemophilia using modified AAV3 vectors.
Capsid- and genome-modified AAV3 vectors for hemophilia gene therapy.
['FUNDING_R01'] · UNIVERSITY OF FLORIDA · NIH-11024765
This study is working on improving gene therapy for hemophilia A and B by creating better delivery systems that help get the right genes into liver cells, which could lead to safer and more effective treatments for people with these bleeding disorders.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF FLORIDA (nih funded) |
| Locations | 1 site (GAINESVILLE, UNITED STATES) |
| Trial ID | NIH-11024765 on ClinicalTrials.gov |
What this research studies
This research focuses on enhancing gene therapy for hemophilia A and B by developing modified adeno-associated virus (AAV3) vectors. The approach involves creating capsid- and genome-modified AAV3 vectors that are more efficient in delivering therapeutic genes to liver cells, which are crucial for producing clotting factors. By optimizing these vectors, the research aims to improve transgene expression and reduce the need for high doses and immune suppression. Patients may benefit from a more effective and safer gene therapy option for their bleeding disorders.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with hemophilia A or B who are seeking innovative treatment options.
Not a fit: Patients with other bleeding disorders not related to hemophilia may not receive benefits from this research.
Why it matters
Potential benefit: If successful, this research could provide a more effective and safer gene therapy for patients with hemophilia, potentially reducing bleeding episodes and improving quality of life.
How similar studies have performed: Previous research has shown success with AAV vectors in gene therapy for hemophilia, but this approach with AAV3 is novel and aims to improve upon existing methods.
Where this research is happening
GAINESVILLE, UNITED STATES
- UNIVERSITY OF FLORIDA — GAINESVILLE, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: SRIVASTAVA, ARUN — UNIVERSITY OF FLORIDA
- Study coordinator: SRIVASTAVA, ARUN
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.
Conditions: bleeding disorder, Blood Coagulation Disorders, Christmas Disease, clotting disorder