Variant-Aware Off-Target Analysis For Informed Guide RNA Selection

To mitigate the risks of off-target activity in CRISPR-based gene editing, it is important to consider the genetic diversity of human populations. By combining computational and biochemical methods, researchers can implement a variant-aware approach to guide RNA selection. This multi-step process allows for the systemic reduction of off-target risk at each stage. For example, in a study focusing on the PCSK9 gene, an in silico tool was used to screen guide RNA candidates, followed by a multiplexed biochemical assay to confirm specificity across various genetic backgrounds. This integrated approach improves confidence in therapeutic genome editing and prepares it for regulatory review.

To learn more about how this variant-aware, multi-step process can be utilized for your therapeutic genome editing workflows, download the poster below.