A new method to assess the safety of genome editing therapies
Official title: Sensitive, unbiased, high-throughput, cellular GUIDE-seq-2 genome-wide activity assay for therapeutic genome editing INDs
Funded at St. Jude Children's Research Hospital · NIH-11093584 · Data updated 2026-11-12
This study is working on a new test to make sure that gene editing treatments are safe and don’t accidentally cause harmful changes in your DNA, so that these therapies can be safer for patients like you.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | St. Jude Children's Research Hospital NIH-funded |
| Lab location | 1 site (Memphis, United States) |
| Project start | June 1, 2023 |
| Project end | May 31, 2026 |
| Funding year | 2025 |
| Award (this year) | $479,100 |
| NIH project number | 5U01AI176470-03 |
| Project ID | NIH-11093584 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are patients with genetic conditions or cancers that may be treated with genome editing therapies.
✕Less likely to help
Patients who do not have genetic disorders or cancers may not receive direct benefits from this research.
What this research studies
This research focuses on developing a refined assay called GUIDE-seq-2, which aims to evaluate the safety and effectiveness of genome editing therapies. By identifying and characterizing unintended mutations that could arise during the editing process, the research seeks to ensure that these therapies do not activate harmful genes. Patients may benefit from this work as it aims to enhance the safety profile of potential gene therapies, making them safer for clinical use. The approach involves optimizing and qualifying this assay for regulatory submissions, ensuring that it meets the necessary standards for therapeutic applications.
Why it matters
Potential benefit: If successful, this research could lead to safer genome editing therapies for patients with genetic disorders or cancers.
How similar studies have gone: Other research has shown success with similar genome editing safety assessments, indicating a promising avenue for improving therapeutic applications.
Where this research is happening
-
St. Jude Children's Research Hospital
Memphis, United States Active
Researchers
- Principal investigator: Tsai, Shengdar — St. Jude Children's Research Hospital
- Study coordinator: Tsai, Shengdar
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.