Using human organoids to evaluate genome editing therapies
Official title: Utility of Human Organoids for Safety and Efficiency Evaluations of Genome Editing Therapeutics
Funded at University of Washington · NIH-11075125 · Data updated 2026-11-12
This study is looking at new ways to test gene editing treatments using tiny 3D models of human organs, like the heart and liver, to see how well they work and if they have any side effects, making it safer for people who might need these therapies.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Washington NIH-funded |
| Lab location | 1 site (Seattle, United States) |
| Project start | May 1, 2023 |
| Project end | April 30, 2026 |
| Funding year | 2025 |
| Award (this year) | $341,841 |
| NIH project number | 5U01AI176460-03 |
| Project ID | NIH-11075125 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals with genetic conditions that may benefit from genome editing therapies.
✕Less likely to help
Patients with conditions that are not targeted by genome editing therapies may not receive any benefit from this research.
What this research studies
This research focuses on developing advanced testing methods for the safety and efficiency of genome editing therapies using human organoids, which are 3D models that mimic human organs. By creating organoids from various human tissues such as kidney, liver, brain, lung, retina, and heart, the researchers aim to assess how well these therapies work and identify any potential side effects. The study will involve optimizing assays to measure the effectiveness of gene editing and comparing results with other research teams to ensure reliability. This approach aims to provide a more accurate representation of human responses compared to traditional animal models.
Why it matters
Potential benefit: If successful, this research could lead to safer and more effective genome editing therapies for patients.
How similar studies have gone: Other research has shown promise in using organoid models for drug testing, indicating that this approach could be effective for genome editing as well.
Where this research is happening
-
University of Washington
Seattle, United States Active
Researchers
- Principal investigator: Freedman, Benjamin Solomon — University of Washington
- Study coordinator: Freedman, Benjamin Solomon
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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.