While CAR therapy has begun to demonstrate efficacy, cell-engineering techniques that result in permanent genomic modification carry several safety concerns. CAR expression driven by RNA creates a platform for delivery of highly-active cell therapy while avoiding long-term CAR-driven toxicity. Using models of pediatric neuroblastoma, we have found that RNA CAR T cell activity is limited by ineffective tumor infiltration.
Keywords: GD2; Lenti CAR, lentivirally-modified chimeric antigen receptor T cell; RNA; RNA CAR, RNA-modified chimeric antigen receptor T cell; chimeric antigen receptors; pediatrics; solid tumors.