Phenotypic correction after WU-106/AMD3100 mobilization and in vivo HSC prime–editing blood analyses. (A) Complete blood cell count using week 16 samples after in vivo transduction (n = 7). Blood samples from healthy CD46.tg mice (n = 3) and untreated CD46/Townes mice (n = 4) were used as the controls. For comparisons of multiple groups, 1-way and 2-way ANOVA with Bonferroni posttest for multiple comparisons was used. Statistical analysis was performed using GraphPad Prism version 10.0.3. ∗∗∗P ≤ .001, ∗∗P ≤ .01, ∗P ≤ .05. (B) Representative microphotographs of the blood cell smears from week 16 samples. First panel: smears of total blood cells subjected to a sickling assay; second panel: blood cell smears stained with Giemsa; third panel: staining of blood smears for reticulocytes with brilliant cresyl blue, which stains nuclear remnants of basophilic ribonucleoproteins in reticulocytes (black arrow). The scale bars are 20 μm. (C) Upper panel: percentage of sickle cells in the blood smears. Each dot represents the percentage in an individual mouse. Lower panel: percentage of reticulocytes in blood smears. (D-F) Comparisons of body weight (D), number of BM MNCs (E), and number of Lin^– cells in untreated and in vivo transduced animals. ns, not significant.