Assessing a single-cell multi-omic analytic platform to characterize ex vivo-engineered T-cell therapy products.

ABSTRACT: Genetically engineered CD8⁺ T cells are being explored for the treatment of various cancers. Analytical characterization represents a major challenge in the development of genetically engineered cell therapies, especially assessing the potential off-target editing and product heterogeneity. As conventional sequencing techniques only provide information at the bulk level, they are unable to detect off-target CRISPR translocation or editing events occurring in minor cell subpopulations. In this study, we report the analytical development of a single-cell multi-omics DNA and protein assay to characterize genetically engineered cell products for safety and genotoxicity assessment. We were able to quantify on-target edits, off-target events, and potential translocations at the target

SUBMITTER: Moshref M 

PROVIDER: S-EPMC11368872 | biostudies-literature | 2024

REPOSITORIES: biostudies-literature