Analysis of translocation events in edited human CD34+ cells
(A) Example of sequences generated by translocation PCR demonstrating dual alignment with both BCL11A-ee and HBG-113. (B) Example of agarose gel electrophoresis image demonstrating presence of translocation product in dual-edited reactions but not single-edited reactions. (C) Frequency of translocation events #1 (HBG F2 + BCL11A R1 primers) and #2 (BCL11A F1 + HBG R2 primers) in vitro by ddPCR (n = 3) (mean ± SD). BCL11A, BCL11A-ee; HBG, HBG-113; Seq, sequential; Sim, simultaneous.