Table 1.
Gene | gRNA Formulation | Alt-R EE Insertion (%) | SD |
---|---|---|---|
RAG2 | Alt-R sgRNA | 0.009 | 0.01 |
Alt-R 2-part XT RNA | 0.013 | 0.01 | |
Alt-R 2-part RNA | 0.035 | 0.03 | |
RAG1 | Alt-R sgRNA | 0.016 | 0.02 |
Alt-R 2-part XT RNA | 0.052 | 0.05 | |
Alt-R 2-part RNA | 0.017 | 0.02 |
The gDNA of CD34+ HSPCs was examined by NGS for the integration of the Alt-R EE, following genome editing with 4 μM RNP complexes, as described in Figure 1. SD, standard deviation.