A New Tool for Creating Better Models of Human Diseases Like Cancer
Site-specific Integration of Large (10-100 kb) DNA Constructs into the Mouse Genome and Human Induced Pluripotent Stem Cells Using the Cas9-Bxb1 Integrase Toolbox
This project is building a powerful new gene-editing tool to help scientists create more accurate models of human diseases, including cancer, using mice and human stem cells.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Jackson Laboratory NIH-funded |
| Lab location | 1 site (Bar Harbor, United States) |
| Project ID | NIH-11109647 on NIH RePORTER |
What this research studies
Current gene-editing methods, like CRISPR-Cas9, are great but struggle to insert very large pieces of DNA into cells, which limits how well we can model complex human diseases. Many diseases, including cancers, involve large genetic changes that are hard to replicate in the lab. This project is developing a special gene-editing toolbox that combines the precision of CRISPR-Cas9 with another system called Bxb1 integrase. This new tool will allow researchers to accurately insert large DNA segments into mouse cells and human stem cells, creating more faithful models of human conditions.
Who could benefit from this research
Good fit: This foundational work does not involve direct patient participation, but future patients with complex genetic diseases, including various cancers, could ultimately benefit from the improved disease models this tool enables.
Not a fit: Patients seeking immediate treatment or direct clinical intervention will not find direct benefit from this basic science tool development project.
Why it matters
Potential benefit: If successful, this new tool will allow scientists to create much more accurate models of human diseases, which is a critical step for understanding these conditions better and developing new treatments.
How similar studies have performed: Initial work has shown that this innovative Cas9-Bxb1 toolbox can precisely integrate DNA constructs up to about 43 kb in length in mice, indicating promising early success for this novel approach.
Where this research is happening
Bar Harbor, United States
- Jackson Laboratory — Bar Harbor, United States (Active)
Researchers
- Principal investigator: Hosur, Vishnu — Jackson Laboratory
- Study coordinator: Hosur, Vishnu
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.