Improving safety in genetic editing by identifying off-target effects
Official title: Robust verification of genetic variant-associated candidate off-target sites
Funded at Boston Children's Hospital · NIH-11145451 · Data updated 2026-11-12
This study is working on making genetic editing treatments safer for everyone by finding and checking for unexpected changes that might happen because of individual genetic differences, using a new tool called CRISPRme and a method called ABSOLVE-seq.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Boston Children's Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project start | May 19, 2025 |
| Project end | April 30, 2028 |
| Funding year | 2025 |
| Award (this year) | $856,513 |
| NIH project number | 1U01AI191210-01 |
| Project ID | NIH-11145451 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals with genetic diseases who may benefit from genome editing therapies and have diverse genetic backgrounds.
✕Less likely to help
Patients who do not have genetic diseases or those whose conditions are not amenable to genetic editing therapies may not receive benefit from this research.
What this research studies
This research focuses on enhancing the safety of genetic editing therapies by identifying potential off-target effects that could arise due to genetic variations among individuals. The team has developed a computational tool called CRISPRme, which predicts off-target sites based on genetic variants, and an experimental method named ABSOLVE-seq to verify these predictions in relevant cell types. By optimizing these tools and integrating them into a comprehensive assessment pipeline, the research aims to ensure that genetic therapies are safer and more effective for diverse patient populations.
Why it matters
Potential benefit: If successful, this research could lead to safer genetic therapies that minimize unintended effects, ultimately improving treatment outcomes for patients with genetic diseases.
How similar studies have gone: Other research has shown promise in improving the safety of genetic editing through similar predictive and verification approaches, indicating a potential for success in this novel application.
Where this research is happening
-
Boston Children's Hospital
Boston, United States Active
Researchers
- Principal investigator: Bauer, Daniel Evan — Boston Children's Hospital
- Study coordinator: Bauer, Daniel Evan
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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.