The advent of CRISPR and other gene editing technologies, which facilitate gene editing in living organisms, has created exciting new possibilities within the life sciences for therapeutic treatments of genetic diseases and cancer immunotherapy. Recent clinical validation, including an early 2020 trial that demonstrated CRISPR’s safety and utility in three cancer patients, has greatly accelerated interest in gene editing-based therapeutics.
Subsequently, governing regulatory bodies — including the FDA — are making on and off target events identification a top priority. To meet evolving product characterization standards, researchers using CRISPR are expected to understand the detailed impact of gene editing on both target and off target sites.
In light of these new expectations, bioinformatics-based off target site predictions and chromosomal translocation events are no longer sufficient. Instead, researchers should prioritize precision analysis using off target-specific testing modalities. Depending on the project requirements, these modalities should utilize rhAmp-seq and targeted amplicon sequencing on Illumina NGS platforms, as well as translocation events using ddPCR assays.