Award
National Institute of Allergy and Infectious Diseases 4U01AI191210-02
Robust verification of genetic variant-associated candidate off-target sites
Recipient
Boston Children's Hospital
Award Amount
$1,675,364.00
Ceiling
$1,675,364.00
Awarded
April 27, 2026
Identifier
4U01AI191210-02
A research grant awarded to Boston Children's Hospital to develop and optimize methods for verifying off-target effects in gene editing therapies, incorporating genetic variant data and experimental validation techniques.
Description
This project aims to optimize computational and experimental methods to assess unintended off-target effects of gene editing therapies, considering human genetic diversity. The approach combines CRISPRme, a tool for nominating off-target sites based on genetic variants, with ABSOLVE-seq, an experimental verification method. The goal is to develop a comprehensive pipeline for assessing off-target risks in genome editing therapeutics, supporting IND applications and regulatory submissions.