Extended Data Fig. 10: In vivo CIM053-SG3376 mediated selective PDX eradication with preserved haematopoiesis of edited cells.

a, Flow cytometry analysis depicting the percentage of human chimerism (%BC8⁺mCD45⁻, blue gate) in the spleen (a) and blood (b) of mice humanised with control (sgNTC) or edited (sg49.3) HSPCs and subsequently treated with either saline solution or CIM053-SG3376. Edited (%BC8⁺QA17A19⁻) and unedited (%BC8⁺QA17A19⁺) cells within the human cell population (%BC8⁺mCD45⁻, blue gate) are also shown (bottom panels). Absolute cell counts of representative panels calculated according to input counting beads are written below the plot. c, Quantification of the human chimerism (PDX versus HSC donor derived cells) in the bone marrow and spleen of mice humanised with control (sgNTC) or shielded (sg49.3) HSPCs, followed by PDX injection, 3 days before saline or CIM053-SG3376 treatment (1-3 mice per group). d, Quantification of the human chimerism (PDX vs HSC donor derived cells) in the spleen of mice from (Fig. 5c). e, Next generation sequencing of spleens of mice from (Fig. 5c). NGS reads accounting for less than 0.8% of total reads were classified as “Others”. (In (a, b, d, e), 1-6 mice depending on the group, data are presented as mean values).

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