Fig. 6: Modified vector with removal of cryptic SA corrects X-SCID CD34+ HSCs. | Nature Communications

Fig. 6: Modified vector with removal of cryptic SA corrects X-SCID CD34+ HSCs.

From: Lentivector cryptic splicing mediates increase in CD34+ clones expressing truncated HMGA2 in human X-linked severe combined immunodeficiency

Fig. 6

Functional restoration of X-SCID CD34+ HSCs transduced by modified lentivector. a Stages of T-cell differentiation using the Artificial thymic organoid. b Percentages of in vitro CD34-derived T-cell progenitors. c Percentages of γ+ cells (by flow cytometry) following in vitro CD34+ differentiation into T cells. d Percentages of γ+ NK cells (by flow cytometry) following in vitro differentiation. e Phospho-STAT5 signaling in in vitro-differentiated T cells derived from transduced X-SCID CD34+ HSCs. f Vector copy number determined by ddPCR in HSCs transduced by clinical lentivector (LV) or the modified vector (LV-modified). For all graphs, data are showed as mean ± standard deviation (SD); n = 2 independent experiments. Source data are provided as a Source Data file.

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