Improving gene therapy for hemophilia A by targeting liver cells for factor VIII production
Official title: Targeted expression of factor VIII in liver sinusoidal endothelial cells for gene therapy for hemophilia A
Funded at Children's Hosp of Philadelphia · NIH-10675697 · Data updated 2026-11-12
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
| 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 start | September 1, 2020 |
| Project end | July 31, 2025 |
| Funding year | 2023 |
| Award (this year) | $641,067 |
| NIH project number | 5R01HL149833-04 |
| Project ID | NIH-10675697 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with hemophilia A who experience frequent bleeding episodes due to low levels of factor VIII.
✕Less likely to help
Patients with other types of bleeding disorders or those who do not have hemophilia A may not benefit from this research.
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.
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 gone: 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
-
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
Related research
Other NIH-funded projects on the same topics.
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cGMP production of FVIII-making placental cells to help people with Hemophilia A
A new, patient-friendly therapy aims to make FVIII-producing placental cells in the lab to help adults with Hemophilia A, potentially offering a longer-lasting and more affordable treatment.
-
A safer, longer-lasting gene delivery approach for Hemophilia A using improved nonviral vectors
Researchers are testing a new type of nonviral gene delivery that could provide longer expression of a therapeutic gene for adults with Hemophilia A.
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Hemophilia A: studying why some people develop inhibitors to FVIII across families
This NIH grant funds a multi-year program to follow families affected by severe hemophilia A to understand why some individuals develop antibodies against factor VIII.
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Intraosseous delivery of gene therapy vectors for hemophilia A
A gene therapy approach using vectors delivered into bone to help people with hemophilia A reduce or eliminate the need for frequent FVIII treatments.
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Targeting FVIII production to eye-opening blood vessels in hemophilia A
Aims to improve treatment for people with hemophilia A by directing how the body makes clotting factor VIII, using a targeted gene-delivery approach.
-
Platelet-targeted FVIII Gene Therapy for Hemophilia A
A gene therapy approach aims to make platelets carry FVIII to help people with hemophilia A, even if they have inhibitors against FVIII.
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.