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. Author manuscript; available in PMC: 2017 Jul 7.
Published in final edited form as: Sci Transl Med. 2016 Oct 12;8(360):360ra134. doi: 10.1126/scitranslmed.aaf9336

Figure 3. Correction of the SCD mutation in SCD HSPCs.

Figure 3

A) Editing the SCD mutation in un-expanded CD34+ HSPCs from the whole blood of SCD patients, assessed by NGS. B) HPLC trace depicting hemoglobin production in SCD HSPCs edited with 200 pmol of the trG10 RNP and the T111-27S donor, compared to untreated HSPCs, after differentiation into erythroblasts. Significant increases in HbA, HbF, and HbA2 are apparent. C) Stacked bars showing HPLC results, with HSPCs edited as indicated after differentiation into erythroblasts. D) Globin gene levels in SCD HSPCs edited as in Fig. 3C, determined by RNA-seq.