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Base editing promises the capacity for precise gene modifications, which could have a substantial impact on the development of therapeutics to treat rare disease and on the next generation of cell-based therapies. For some cell therapies to be successful, such as the use of chimeric antigen receptor (CAR) T cells in the treatment of solid tumors, multiple gene engineering events will be required.
To date, substantial improvements to the efficacy of CAR-T cells have come from altering the design of the CAR resulting in the improved activation of the T cell. But for the solid tumor microenvironment, additional genetic changes to the T cell will be required, such as modulating genes that regulate survival and persistence in a hostile environment and reducing the onset of exhaustion. Compared with currently-available gene editing methodologies, such as CRISPR–Cas9, which creates a double strand break in order to edit the DNA potentially leading to unintended off-target effects, base editing is a more precise gene engineering technology that relies on ‘nicking’ one DNA strand to effect change in the DNA sequence.