Article | June 1, 2022

Genome Editing Using mRNA-Lipid Nanoparticles For CAR T Cell Therapy

GettyImages-970394878-genetic-engineering-gene-editing-dna

The development of genome editing technologies has provided scientists with ways to directly target and modify the genomic sequences of a living organism. It has extended our understanding of the genetics behind human disease by enabling the creation of more accurate cellular and animal models. However, delivery of gene editing tools to target cells has been a challenge. New technologies for safe and efficient gene delivery that can overcome payload limitations will enable researchers to realize the full potential of gene editing strategies to explore new avenues for cancer treatment and beyond.

Gene engineering of T cells to produce new cancer immunotherapies like chimeric antigen receptor (CAR)-T cell therapy, have revolutionized cancer treatment. Current autologous CAR-T immunotherapies have demonstrated high clinical success, but there is a need to improve safety and efficacy profiles. Next generation CAR-T designs focus on enhancing CAR-T cell potency, limiting off-target effects, broadening the therapeutic targets beyond liquid cancers, and manufacturing universal CAR-T cells from allogeneic donors1. These new strategies require more complex CRISPR/Cas9-enabled genetic engineering strategies where the chosen gene delivery method plays a central role in determining the gene editing efficiency, safety, and scalability.

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