Large Serine Integrase Off-target Discovery and Validation for Therapeutic Human Genome Editing

Time: 9:00 am
day: Conference Day Two


  • Large serine integrases (LSIs) have emerged as an attractive therapeutic modality for the site specific genomic integration of DNA because they are sequence specific, size independent and do not rely on endogenous DNA repair mechanisms
  • New technologies and computational methods that account for all potential unintended genome editing related to LSIs are required to advance their clinical application for therapeutic gene integration
  • Compared to whole genome sequencing on a population of edited cells, the combination of fit-for-purpose discovery technologies and validation assays with hybrid capture sequencing is 45-fold more sensitive