Using ultrasound to deliver genes for treating hemophilia A
Official title: Ultrasound-mediated gene delivery to achieve therapeutic correction of hemophilia A
Funded at Seattle Children's Hospital · NIH-10599134 · Data updated 2026-11-12
This study is exploring a new way to help people with hemophilia A by using ultrasound to safely deliver genes that can help their bodies make the missing factor VIII protein, which could lead to better and more affordable treatments.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Seattle Children's Hospital NIH-funded |
| Lab location | 1 site (Seattle, United States) |
| Project start | April 1, 2020 |
| Project end | March 31, 2026 |
| Funding year | 2023 |
| Award (this year) | $774,217 |
| NIH project number | 5R01HL151077-04 |
| Project ID | NIH-10599134 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with hemophilia A, particularly those who may not respond well to current therapies.
✕Less likely to help
Patients with other forms of hemophilia or those who do not have a deficiency of factor VIII may not benefit from this research.
What this research studies
This research aims to develop a noninvasive method using ultrasound to deliver genes that can correct the deficiency of factor VIII in hemophilia A. By using ultrasound-mediated gene delivery, the researchers hope to provide a more effective and less costly treatment option compared to current protein replacement therapies. The approach focuses on delivering plasmid vectors that can produce therapeutic levels of factor VIII protein, potentially offering long-term correction of the disorder. This method is designed to minimize immune responses and allow for repeated treatments if necessary.
Why it matters
Potential benefit: If successful, this research could provide a long-lasting and cost-effective treatment for patients with hemophilia A.
How similar studies have gone: Previous clinical trials using viral gene therapy for hemophilia A have shown promising results, indicating that gene therapy approaches are viable, though this ultrasound-mediated method is relatively novel.
Where this research is happening
-
Seattle Children's Hospital
Seattle, United States Active
Researchers
- Principal investigator: Miao, Carol H — Seattle Children's Hospital
- Study coordinator: Miao, Carol H
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.
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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.
-
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.