Improving gene therapy for hemophilia A by targeting liver cells for factor VIII production
Targeted expression of factor VIII in liver sinusoidal endothelial cells for gene therapy for hemophilia A
['FUNDING_R01'] · CHILDREN'S HOSP OF PHILADELPHIA · NIH-10675697
This study is testing a new gene therapy for people with hemophilia A to help their bodies make more of the factor VIII protein they need to stop bleeding, using a different method that targets specific liver cells, which could lead to better treatment results.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | CHILDREN'S HOSP OF PHILADELPHIA (nih funded) |
| Locations | 1 site (PHILADELPHIA, UNITED STATES) |
| Trial ID | NIH-10675697 on ClinicalTrials.gov |
What this research studies
This research focuses on developing a gene therapy approach for hemophilia A, a bleeding disorder caused by a deficiency in coagulation factor VIII. The study aims to enhance the production of factor VIII by targeting liver sinusoidal endothelial cells instead of the traditionally targeted hepatocytes. By using adeno-associated viral vectors, the researchers hope to achieve higher levels of factor VIII expression, which could lead to better management of the disease. Patients with hemophilia A may benefit from this innovative approach that seeks to improve treatment outcomes significantly.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with hemophilia A who experience frequent bleeding episodes due to low levels of factor VIII.
Not a fit: Patients with other types of bleeding disorders 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 more effective and durable treatments for patients with hemophilia A, reducing bleeding episodes and improving quality of life.
How similar studies have performed: While gene therapy for hemophilia A is being explored in clinical trials, this specific approach targeting liver sinusoidal endothelial cells is novel and has not been extensively tested.
Where this research is happening
PHILADELPHIA, UNITED STATES
- CHILDREN'S HOSP OF PHILADELPHIA — PHILADELPHIA, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: SABATINO, DENISE E — CHILDREN'S HOSP OF PHILADELPHIA
- Study coordinator: SABATINO, DENISE E
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: Blood Coagulation Disorders, Coagulation Disorder, bleeding disorder, clotting disorder