Using ultrasound to deliver genes for treating hemophilia A
Ultrasound-mediated gene delivery to achieve therapeutic correction of hemophilia A
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 ID | NIH-10599134 on NIH RePORTER |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with hemophilia A, particularly those who may not respond well to current therapies.
Not a fit: Patients with other forms of hemophilia or those who do not have a deficiency of factor VIII may not benefit from this research.
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 performed: 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, United States
- Seattle Children's Hospital — Seattle, United States (Active)
Researchers
- Principal investigator: Miao, Carol H — Seattle Children's Hospital
- Study coordinator: Miao, Carol H
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.