Building on a New Approach to Validate Gene Editing and Localize CAR Cassettes in T Cells
Safety and efficacy are key considerations in engineered cell therapies. Any technology that modifies the genome has the potential to cause genotoxicity. The accuracy of editing and the potential occurrence of unintended rearrangements should be assessed to understand the associated risks, such as cassette insertion in the vicinity of an oncogene or a tumor suppressor. However, conventional PCR screening for editing outcomes can overlook such unintended insertions. Here, we discuss a workflow using the Xdrop system that is proven to overcome this limitation to identify the precise localization of the CAR insertion. The workflow requires just 10 ng of DNA as starting material, making it relevant during engineered cell development, after cell expansion, for post-transfusion monitoring, and in unexpected clinical event investigation.
