Targeted DNA insertion in human cells using a piggyBac-based platform
Official title: Development of a Targetable Transposase Platform for Precision Gene Integration in Human Cells
Funded at University of Hawaii at Manoa · NIH-11406919 · Data updated 2026-11-12
A new platform aims to safely and precisely insert large pieces of DNA into human cells without relying on risky DNA breaks, with potential for gene therapies and research.
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 start | September 8, 2025 |
| Project end | June 30, 2029 |
| Funding year | 2026 |
| Award (this year) | $495,282 |
| NIH project number | 5R01GM160155-02 |
| Project ID | NIH-11406919 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates include adults with conditions that could benefit from future gene-therapy approaches using precise DNA insertion.
✕Less likely to help
Patients without diseases that would utilize gene insertion therapies may not benefit directly.
What this research studies
This grant focuses on building a piggyBac transposase-based system to insert large DNA fragments into specific spots in the human genome, addressing limitations of current CRISPR methods in non-dividing cells. The approach uses design and evolution techniques to improve targeting and reduce off-target effects, aiming for a safe, efficient tool for therapeutic gene delivery. The work includes testing in human cells to demonstrate precise integration and minimal unintended changes, moving toward future clinical applications.
Why it matters
Potential benefit: If successful, patients could benefit from more reliable gene therapies that require precise insertion of therapeutic DNA with fewer side effects.
How similar studies have gone: Similar targeted integration approaches have shown promise, but this platform seeks to improve precision and safety for large DNA insertions.
Where this research is happening
-
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
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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.