Developing tools for precise genome editing
Official title: Computational tools for precision genome editing
Funded at Boston Children's Hospital · NIH-11138708 · Data updated 2026-11-12
This study is working on new tools to make genome editing safer and more effective for medical treatments, helping doctors better predict how these changes might affect your DNA.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Boston Children's Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project start | July 15, 2024 |
| Project end | April 30, 2028 |
| Funding year | 2025 |
| Award (this year) | $751,289 |
| NIH project number | 5R01HG013618-02 |
| Project ID | NIH-11138708 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals with genetic conditions that could potentially be treated through genome editing, such as beta-thalassemia.
✕Less likely to help
Patients with conditions that are not amenable to genome editing or those who do not have genetic mutations may not benefit from this research.
What this research studies
This research focuses on creating advanced computational tools to improve the safety and effectiveness of genome editing technologies. It aims to enhance the prediction of both on-target and off-target effects, which are crucial for the clinical application of these technologies. The project will refine existing tools and develop new ones to better understand the complexities of DNA modifications, ensuring that genome editing can be applied safely in medical treatments. By leveraging diverse human genomes and advanced sequencing methods, the research seeks to provide a clearer picture of how genome editing can be optimized for patient care.
Why it matters
Potential benefit: If successful, this research could lead to safer and more effective genome editing therapies for patients with genetic disorders.
How similar studies have gone: Previous research has shown promise in developing computational tools for genome editing, indicating that this approach has the potential for significant advancements.
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.