Evaluating the safety and effectiveness of genome editing in human cells
Official title: Genome sequencing for evaluating the efficacy, specificity, and safety of human genome editing
Funded at Washington University · NIH-11056729 · Data updated 2026-11-12
This study is looking at how well gene editing works in human cells and making sure it's safe, so that patients can have better and safer treatments in the future.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Washington University NIH-funded |
| Lab location | 1 site (Saint Louis, United States) |
| Project start | April 20, 2023 |
| Project end | March 31, 2026 |
| Funding year | 2025 |
| Award (this year) | $458,717 |
| NIH project number | 5U01AI176465-03 |
| Project ID | NIH-11056729 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are patients undergoing cellular therapies that involve genome editing, particularly those with genetic disorders or cancers.
✕Less likely to help
Patients who are not receiving genome-edited therapies or those with conditions unrelated to genetic mutations may not benefit from this research.
What this research studies
This research focuses on using whole-genome sequencing to assess how well genome editing works in human cells and to ensure its safety. By analyzing the genetic changes made during the editing process, the researchers aim to identify both intended and unintended mutations that could affect patient outcomes. This comprehensive approach is designed to meet FDA guidelines and improve the reliability of cellular therapies that utilize genome editing. Patients may benefit from more effective and safer treatments as a result of this research.
Why it matters
Potential benefit: If successful, this research could lead to safer and more effective genome-edited therapies for various genetic conditions and cancers.
How similar studies have gone: Previous research has shown promise in using whole-genome sequencing for evaluating genome editing, indicating that this approach could be effective.
Where this research is happening
-
Washington University
Saint Louis, United States Active
Researchers
- Principal investigator: Spencer, David H — Washington University
- Study coordinator: Spencer, David H
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.