Gates Package — page 851

Fig.8. -globin analysis after in vivo base editing with by HDAd-EF1a.ABE8e. A) - globin expression in peripheral RBCs measured by flow cytometry. N=5 animals. B) - globin expression at mRNA level measured by qRT-PCR. Data shown are fold of change over mouse HBA, mouse HBB, or human HBB mRNA. N=5 animals. C) Human -globin chain levels in RBC lysates measured by HPLC. Data shown are percentages over mouse a- or β-globin or human β-globin. N=5 animals. D, E) Immunofluorescence analysis of HbF in erythrocytes. D) Human samples. Cytospins of total blood cells from umbilical cord blood (fetus-derived) and from an adult donor. E) Samples from untreated and treaded -YAC mice (total blood). Cytospins were stained with a GFPspecific antibody. Shown are representative samples out of 5 cytospins per group. The scale bar is 20mm.