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Cas9 variant gene editing of the HBB locus with a ssODN donor. CD34+ HSPCs edited with Cas9 variants (3 pmol Cas9 mRNA + 120 pmol IVT sgRNA) and ssODN donor (3 μM). Editing outcomes were measured by MiSeq HTS 4 days post-electroporation. Cas9 and Cas9-hGem were used as controls. (A) HDR and NHEJ. (B) HDR/NHEJ n = 2 biological replicates. Error bars, mean ± SD. (C) MMEJ/indels. n = 2 biological replicates. Center line represents mean.
