Improving gene editing to create better models of human diseases
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 study is working on a new way to help scientists insert large pieces of DNA into the genes of mice and human stem cells, which will make it easier to understand and model diseases like cancer.
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-10886696 on NIH RePORTER |
What this research studies
This research focuses on enhancing the ability to integrate large DNA segments into the genomes of mice and human stem cells, which is crucial for accurately modeling human diseases like cancer. The team is developing a new gene-editing toolbox that combines the precision of CRISPR-Cas9 with a specialized integrase called Bxb1, allowing for the efficient insertion of large DNA constructs. This innovative approach aims to overcome current limitations in genetic engineering, enabling researchers to study complex genetic variations associated with diseases more effectively.
Who could benefit from this research
Good fit: Ideal candidates for participation or benefit from this research include individuals with genetic conditions that could be modeled in mice or stem cells, particularly those related to cancer.
Not a fit: Patients with conditions that do not have a genetic basis or are not related to the diseases being modeled may not receive any benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to more accurate models of human diseases, improving our understanding and treatment of conditions like cancer.
How similar studies have performed: Other research has shown success with similar CRISPR-based approaches, but the specific integration of large DNA constructs using the Cas9-Bxb1 toolbox is a novel and untested method.
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.