Testing personalized treatments using patient-derived mouse models
Preclinical/Co-Clinical Section
Researchers use mouse models made from patient samples to test gene-targeting therapies and repurposed drugs for people with specific genetic 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 ID | NIH-11248593 on NIH RePORTER |
What this research studies
This program creates mouse models that reflect an individual patient's disease using samples and clinical information gathered with clinical partners. Scientists compare proteins and other molecular features between patients and the mice to find markers and disease mechanisms. Based on those findings, they design genome-based therapies like antisense oligonucleotides, gene replacement, siRNA, or try repurposed drugs and screen them in cells and in mice. Successful candidates are advanced toward the data package needed before human trials.
Who could benefit from this research
Good fit: People with specific genetic mutations or rare genetic conditions who can share samples and clinical data with the research team would be ideal candidates to support this work.
Not a fit: Patients without a matching genetic change or whose condition is not modeled by the program are unlikely to receive direct benefit from this grant.
Why it matters
Potential benefit: If successful, this work could speed development of targeted treatments and biomarkers that move some genetic-disease therapies closer to clinical trials.
How similar studies have performed: Patient-derived models and antisense oligonucleotides have led to successful therapies in some genetic diseases, so this approach builds on existing, promising methods.
Where this research is happening
Bar Harbor, United States
- Jackson Laboratory — Bar Harbor, United States (Active)
Researchers
- Principal investigator: Lutz, Cathleen M — Jackson Laboratory
- Study coordinator: Lutz, Cathleen M
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.