Eira Leinonen

This paper presents a non-viral HDR-based CRISPR/Cas9 protocol for editing primary T cells with up to 80% HDR efficiency, targeting small pathogenic variants in patient and healthy donor cells.

This paper presents a T cell gene correction pipeline using CRISPR/Cas9 and HDR to correct monogenic mutations in IEI models, achieving up to 80% bi-allelic editing with no detectable off-target effects.