A Novel RNA Lipid Nanoparticle Platform: Gene-Edited CAR T Cells For Off-The-Shelf Cancer Therapy

Engineering T cells with expressions of chimeric antigen receptor (CAR) redirect these cells to target tumours, making this a promising cell-based cancer therapy [1]. To engineer universal CAR T cells for allogeneic cell therapy, gene editing approaches [2] can be used to remove risks associated with graft-versus-host disease [3].

Complex gene editing in primary immune can be challenging for some conventional methods [4]. The LNPs use endogenous uptake pathways to deliver therapeutic RNAs, making this technology gentle to the cells.

Here we report on the use of a novel lipid nanoparticle (LNP) reagent in a validated protocol to achieve successful complex gene editing in primary T cells with high efficiency while maintaining high cell viability.