Award
NIH Reporter #5R01HG013618-03
Computational tools for precision genome editing
Recipient
Boston Children's Hospital
Award Amount
$751,288.00
Ceiling
$751,288.00
Awarded
August 21, 2026
Identifier
5R01HG013618-03
The award funds the development of computational tools to improve the precision and safety of genome editing technologies, including software development and dataset generation to study CRISPR experiments.
Description
This project proposes to develop and refine computational tools to advance the safety and efficacy of genome editing, addressing the critical need for precise quantification and prediction of on- and off-target effects. Genome editing technologies have the potential to revolutionize the treatment and prevention of disease by enabling precise modifications to DNA sequences. However, their clinical application is currently limited by significant challenges in detecting and avoiding off-target effects, as well as quantifying and understanding the heterogeneity of on-target edits. The project includes developing new tools such as CRISPRus and DE-CRISPR, characterizing complex editing outcomes, and creating benchmark datasets to improve the safety and effectiveness of genome editing therapies.