Using mouse models to test new gene therapies
Official title: Preclinical Mouse Model Core
Funded at Jackson Laboratory · NIH-11089344 · Data updated 2026-11-12
This study is using mice to learn more about diseases and to try out new treatments, especially those that involve changing genes, so we can eventually help people with these conditions.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Jackson Laboratory NIH-funded |
| Lab location | 1 site (Bar Harbor, United States) |
| Project start | May 16, 2023 |
| Project end | April 30, 2028 |
| Funding year | 2025 |
| Award (this year) | $656,033 |
| NIH project number | 5U19NS132304-03 |
| Project ID | NIH-11089344 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals with genetic conditions that could potentially be treated with gene editing therapies.
✕Less likely to help
Patients with conditions that are not amenable to gene therapy or those who do not have a genetic basis for their disease may not benefit from this research.
What this research studies
This research focuses on utilizing mouse models to better understand disease mechanisms and to test new therapeutic approaches, particularly those involving gene editing. By employing advanced techniques like CRISPR/Cas9, the project aims to validate the efficacy of these treatments in a living organism, which is crucial for moving towards human clinical trials. The Jackson Laboratory provides a centralized resource for these models, ensuring that researchers have access to the necessary tools and expertise for their specific disease studies.
Why it matters
Potential benefit: If successful, this research could lead to the development of effective gene therapies for various diseases, improving treatment options for patients.
How similar studies have gone: Previous research using similar mouse model approaches has shown success in validating gene therapies, indicating a promising avenue for future treatments.
Where this research is happening
-
Jackson Laboratory
Bar Harbor, United States Active
Researchers
- Principal investigator: Murray, Stephen a — Jackson Laboratory
- Study coordinator: Murray, Stephen a
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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.