Carolina Wieczorek Ervik

This paper edits the ADA2 p.R169Q mutation in human hematopoietic stem cells using CRISPR-Cas9 and HDR, showing improved efficiency with NHEJ inhibitors but increased chromosomal deletions.

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.