Improving safety in genetic editing by identifying off-target effects
Robust verification of genetic variant-associated candidate off-target sites
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 ID | NIH-11145451 on NIH RePORTER |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals with genetic diseases who may benefit from genome editing therapies and have diverse genetic backgrounds.
Not a fit: 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.
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 performed: 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, 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.