Figure 1.

CRISPR/Cas9 gene editing and autologous transplantation of RMs. (A) Schematic diagram of an RM CRISPR/Cas9 gene editing/autologous transplantation model. RM HSPCs were mobilized into the blood with granulocyte colony-stimulating factor (G-CSF) and plerixafor (AMD3100). CD34⁺ cells were electroporated with ribonucleoprotein (RNP) complexes containing gRNAs targeting either a PIG-A exon or the AAVS1 safe harbor site and Cas9 protein. Edited cells were reinfused into the autologous macaque after total body irradiation. Long-term tracking of mutated cells was performed by targeted deep sequencing and flow cytometry. (B) Before transplantation, initial editing efficiency of PIG-A and AAVS1 in an infusion product was determined in both RMs by targeted deep sequencing. (C) Transplantation characteristics of RMs included in the study.