Developing a new mouse model for gene therapy evaluation
Official title: Illuminating gene therapy
Funded at University of California, San Francisco · NIH-10752645 · Data updated 2026-11-12
This study is working on a new type of lab mouse that helps scientists better understand how gene therapy works, making it easier to measure its effects and compare results, which could lead to improved treatments for people with genetic conditions.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California, San Francisco NIH-funded |
| Lab location | 1 site (San Francisco, United States) |
| Project start | December 15, 2022 |
| Project end | November 30, 2025 |
| Funding year | 2024 |
| Award (this year) | $242,250 |
| NIH project number | 5R21OD034078-02 |
| Project ID | NIH-10752645 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for benefiting from this research are individuals with genetic conditions that may be treated through gene therapy.
✕Less likely to help
Patients with conditions that are not amenable to gene therapy or those who do not have genetic disorders may not receive any benefit from this research.
What this research studies
This research focuses on creating an innovative mouse model that allows for the quantification of gene therapy effects. By utilizing a multiplexed reporter system, the project aims to enhance the accuracy and reproducibility of gene editing assessments. The model incorporates various advanced technologies, including near-infrared fluorescence proteins and luciferases, to provide multiple measurement options for researchers. This approach not only reduces the number of animals needed for experiments but also facilitates better comparisons across different studies.
Why it matters
Potential benefit: If successful, this research could significantly improve the evaluation and effectiveness of gene therapies, leading to better treatment options for patients with genetic disorders.
How similar studies have gone: While this approach is novel and has not been previously tested, similar methodologies in gene therapy have shown promise in enhancing treatment efficacy.
Where this research is happening
-
University of California, San Francisco
San Francisco, United States Active
Researchers
- Principal investigator: Mcmanus, Michael T — University of California, San Francisco
- Study coordinator: Mcmanus, Michael T
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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.