Extended Data Fig. 2: Off-target analysis of HBA1-specific sg5. | Nature Medicine

Extended Data Fig. 2: Off-target analysis of HBA1-specific sg5.

From: Gene replacement of α-globin with β-globin restores hemoglobin balance in β-thalassemia-derived hematopoietic stem and progenitor cells

Extended Data Fig. 2: Off-target analysis of HBA1-specific sg5.

a, Summary of rhAmpSeq targeted sequencing results at on-target and 40 most highly predicted off-target sites by COSMID for HBA1 sg5. Values are indel frequency for RNP treatment after subtraction of indel frequency for Mock treatment at each locus for each experimental replicate. N = 3 biologically independent HSPC donors, though not all values are displayed since some were <0.01% after subtraction of Mock indel frequencies. Bars represent median. b, List of genomic coordinates for forty most highly predicted off-target sites by COSMID for HBA1 sg5.

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