Fig. 8: Long term engraftment of ABE8e multiplex edited CD90+ HSPCs in rhesus macaques.
From: Multiplex base editing to protect from CD33 directed drugs for immune and gene therapy

ABE8e editing efficiency at the CD33 (a) and HBG (b) targets measured in the infusion product of each transplanted animal at 5 days post multiplex editing or post mock electroporation (unedited, UE). c, Frequency of unedited, edits at each gene target, or at both targets in colony-forming cells (CFCs) obtained from infusion products from a and b. n = 71 for A18031 and n = 37 for A18038. d Frequency of colony-forming cells with unedited (UE), monoallelic (mono) or biallelic (bi) CD33 edits, and accompanying edits (0 to 4) at the HBG target site in the two infusion products. Number of colonies analyzed is shown on top. e Tracking of CD33 and HBG editing in peripheral blood of both transplanted animals. For simplicity, only editing efficiency at the relevant adenines are shown (A7 for CD33 and A5 for HBG). f CD33 expression in CD11b+CD14− peripheral blood granulocytes in both transplanted animals as compared to an untransplanted control animal (A16227). g, Fetal hemoglobin (HbF) expression as measured by flow cytometry staining for F-cells in peripheral blood of both transplanted animals as compared to the untransplanted control. h Colony forming cells (CFCs) derived from BM aspirates taken from both transplanted animals at 8- or 6-months post-transplant for A18031 and A18038, respectively. i, Frequency of unedited, edits at each gene target, or at both targets in CFCs from g n = 57 for A18031 and n = 45 for A18038. All error bars in this figure show ±SEM. Source data are provided as a Source Data file.