Eero Tölö

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.

The paper evaluates DNA repair protein-Cas9 fusions for improving CRISPR editing efficiency, finding that most require optimization for specific cell types and genomic loci.