Value Proposition:
· Utilizes components of existing workflow to better detect off-target gene editing
· Capable for use in in vivo or in vitro settings
· Maximizes impact of CRISPR-Cas9 in therapeutic development
Technology Description
· Researchers at Johns Hopkins have developed a method to detect off-target editing with high sensitivity following CRISPR-Cas9 genetic editing. By elongating the duration of MRE11 localization, a critical component of CRISPR-Cas9 editing, subsequent ChIP-seq allows for detection of all CRISPR-Cas9 binding sites. Off-target sites can then be distinguished from intended targets via results obtained from the MRE11 ChIP-seq.
Unmet Need
· CRISPR-Cas9 is a gene editing technology that has revolutionized therapeutics for genetic diseases such as cystic fibrosis, muscular dystrophy, hemophilia, and more. However, the technology is limited by potential off-target effects. Current methods of off-target detection are limited and either have low sensitivity or cannot be performed directly in vivo. Therefore, there is a need to develop a system that can more accurately detect off-target effects to maximize the impact of gene editing for genetic disease therapies.
Stage of Development
· Researchers have conducted in vivo experiments.
Data Availability
Data available in the publication below.
Publication
Zou, R.S. et al. Improving the sensitivity of in vivo CRISPR off-target detection with DISCOVER-Seq+. Nat Methods
