ONE-seq^® For Variant-Aware Therapeutic Guide Selection

The ONE-seq platform is a powerful tool for identifying and prioritizing potential off-target sites in CRISPR-Cas9 gene editing experiments. It leverages computational tools and biochemical assays to nominate candidate off-target sites across thousands of genomes with high sensitivity. In this study, we applied ONE-seq in a variant-aware manner to nominate off-target sites across global human populations for three therapeutically relevant guide RNAs targeting the PCSK9 gene.

Our results show that ONE-seq can effectively identify off-target sites that are specific to certain populations. This information can be used to select guide RNAs that are less likely to cause off-target editing in specific populations. We also show that ONE-seq can be used to screen multiple guides in a single run, which can reduce the cost and time required for guide selection.

Download our poster to learn more about our findings and the implications for variant-aware guide selection.