Using gene editing and lipid nanoparticles to treat hemophilia A
Official title: Gene Editing for Hemophilia A Treatment Using Lipid Nanoparticles
Funded at Seattle Children's Hospital · NIH-11163538 · Data updated 2026-11-12
This study is testing a new way to treat hemophilia A by using tiny particles to deliver gene-editing tools that could fix the gene causing the bleeding disorder, aiming to offer a more lasting solution than the usual 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 | August 15, 2024 |
| Project end | May 31, 2028 |
| Funding year | 2025 |
| Award (this year) | $734,329 |
| NIH project number | 5R01HL169793-02 |
| Project ID | NIH-11163538 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with hemophilia A who have a deficiency in the factor VIII gene.
✕Less likely to help
Patients with hemophilia B or other bleeding disorders unrelated to factor VIII may not benefit from this research.
What this research studies
This research aims to develop a new treatment for hemophilia A, a bleeding disorder caused by a deficiency in the factor VIII gene. By utilizing lipid nanoparticles to deliver CRISPR/Cas9 gene editing tools, the project seeks to correct the faulty gene responsible for this condition. Current treatments involve frequent and costly infusions of factor VIII protein, which are not always effective. This innovative approach could provide a more permanent solution by directly editing the gene in the patient's liver cells, potentially improving their quality of life.
Why it matters
Potential benefit: If successful, this research could lead to a long-lasting treatment for hemophilia A, reducing the need for frequent infusions and improving patient outcomes.
How similar studies have gone: Previous trials using adeno-associated viral gene therapy for hemophilia A have shown promising results, indicating that gene therapy approaches may be effective.
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.
-
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.