Figure 5.
GE in XMEN T cells. (A) Representative western blot for MAGT1 protein expression in HD XMEN (naive and gene edited) T cells with β-actin as loading control. (B) Percentage of NKG2D-positive CD8+ T cells in AAV-treated T cells at days 2 and 35 post-EP (data are representative of 8 independent experiments; XMEN patients P1 and P2). (C) Targeted insertion rates measured at day 2 and day 35 post-EP (data are representative of 8 independent experiments). (D) Representative flow cytometry histogram overlaying NKG2D expression in HD and XMEN (naive and gene-edited) T cells at day 35 after GE. Percentage of NKG2D+ in gene-edited CD8+ XMEN T cells was 57.5% and TI was 64.6%. (E) Expression of CD70 and CD28 by flow cytometry for HD and XMEN (naive and gene-edited) T cells at day 28 post-EP; data are expressed as the ratio of MFI normalized to XMEN untreated cells for CD70 and CD28 (data are representative of 2-3 independent experiments). A paired sample Student t test was used. ***P < .001; ****P < .0001.

GE in XMEN T cells. (A) Representative western blot for MAGT1 protein expression in HD XMEN (naive and gene edited) T cells with β-actin as loading control. (B) Percentage of NKG2D-positive CD8+ T cells in AAV-treated T cells at days 2 and 35 post-EP (data are representative of 8 independent experiments; XMEN patients P1 and P2). (C) Targeted insertion rates measured at day 2 and day 35 post-EP (data are representative of 8 independent experiments). (D) Representative flow cytometry histogram overlaying NKG2D expression in HD and XMEN (naive and gene-edited) T cells at day 35 after GE. Percentage of NKG2D+ in gene-edited CD8+ XMEN T cells was 57.5% and TI was 64.6%. (E) Expression of CD70 and CD28 by flow cytometry for HD and XMEN (naive and gene-edited) T cells at day 28 post-EP; data are expressed as the ratio of MFI normalized to XMEN untreated cells for CD70 and CD28 (data are representative of 2-3 independent experiments). A paired sample Student t test was used. ***P < .001; ****P < .0001.

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