Improving gene delivery to treat genetic diseases.
Directed evolution of a sequence-specific targeting technology for therapeutic gene delivery to the human genome.
This study is exploring a new way to safely deliver helpful genes to the right spots in our DNA, specifically looking at treating hemophilia by getting the Factor IX gene into the liver of mice, which could lead to better and safer treatments for genetic conditions.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Hawaii at Manoa NIH-funded |
| Lab location | 1 site (Honolulu, United States) |
| Project ID | NIH-10769780 on NIH RePORTER |
What this research studies
This research focuses on developing a new method for safely delivering therapeutic genes to specific locations in the human genome. By evolving integrase enzymes, the team aims to create a system that can insert DNA at precise sites, reducing the risks associated with traditional gene therapy methods. The approach will be tested using a model of hemophilia, where the goal is to deliver the Factor IX gene to the liver of mice. This innovative technique could lead to more effective and safer treatments for genetic disorders.
Who could benefit from this research
Good fit: Ideal candidates for this research include individuals with genetic disorders, particularly those related to Factor IX deficiency or hemophilia.
Not a fit: Patients with conditions unrelated to genetic mutations or those who do not have hemophilia may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could provide a safer and more efficient way to treat genetic diseases by ensuring precise gene delivery.
How similar studies have performed: Other research has shown promise in using directed evolution for gene delivery, suggesting that this approach could lead to significant advancements in the field.
Where this research is happening
Honolulu, United States
- University of Hawaii at Manoa — Honolulu, United States (Active)
Researchers
- Principal investigator: Owens, Jesse Bruce — University of Hawaii at Manoa
- Study coordinator: Owens, Jesse Bruce
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.