Checking gene-editing off-targets tied to genetic differences
Robust verification of genetic variant-associated candidate off-target sites
This work improves tools that check whether gene-editing treatments might accidentally change the wrong places in DNA for people with different genetic backgrounds.
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 ID | NIH-11333023 on NIH RePORTER |
What this research studies
This project combines a computer program called CRISPRme with a lab test named ABSOLVE-seq to find and confirm sites where gene-editing tools might mistakenly cut DNA. The team will add genetic data from the All of Us Research Program and other large datasets so the tools consider real human variation. They will also refine lab steps, vector design, and data analysis to make the tests more sensitive, reliable, and scalable across different editing methods. The goal is a single pipeline that can be used to support safety reviews for gene-editing treatments.
Who could benefit from this research
Good fit: People who might benefit include patients who are candidates for gene-editing therapies or who have genetic conditions that could be treated with genome editing.
Not a fit: Patients not considering gene-editing treatments or whose care does not involve DNA-targeting therapies are unlikely to see direct benefits from this project.
Why it matters
Potential benefit: If successful, this work could make gene-editing treatments safer by identifying patient-specific off-target risks before therapy.
How similar studies have performed: Previous tools and lab assays have detected off-target edits, but combining variant-aware prediction with experimental confirmation across diverse human genomes is relatively new.
Where this research is happening
Boston, United States
- Boston Children's Hospital — Boston, United States (Active)
Researchers
- Principal investigator: Bauer, Daniel Evan — Boston Children's Hospital
- Study coordinator: Bauer, Daniel Evan
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 below.