Anti-Cas9 expressions were similar in pre- and post-transplantation periods for BCL11A enhancer-edited animals
(A) Schematic representation of CRISPR-edited CD34+ HSPC transplantation. Note: The schema was made using biorender.com. (B and C) Anti-Cas9 antibody levels were comparable before and after infusion of BCL11A enhancer (+58 site) and AAVS1, (D and E) BCL11A enhancer (+58 site), or (F–H) BCL11A enhancer (+55 and +58 sites) SpCas9-ribonucleaprotein (RNP)-edited CD34+ HSPC transplanted animals. G-CSF, granulocyte colony stimulating factor; RBC, red blood cell; TBI, total body irradiation; BSA, bovine serum albumin; HSA, human serum albumin. Measles antigens were used as positive control.