Successful ex vivo transduction of CD34+ cells from patients with X-linked severe combined immunodeficiency disease (X-SCID). (IMAGE)

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Successful ex vivo transduction of CD34+ cells from patients with X-linked severe combined immunodeficiency disease (X-SCID).

Caption

(A) CD132 expression in IL2RG defect or corrected CD34+ cells at 2 days and 7 days after transduction. (B) MFI of CD132 in CD34+ cells. (C) Mean VCNs (± standard deviation) in the bulk population of transduced CD34+ cells. (D) The total numbers of colony-forming units (CFUs) of CD34+ cells in both groups (left) and the numbers of colonies of different lineages (right). The light grey indicates the isotype control (iso), the dark grey indicates the healthy control (HC), and the red indicates patients (P). The data were presented as mean ± standard error of the mean. Statistical differences are expressed as follows: ns, non-significant; ∗∗p < 0.01. VCN, vector copy number; MFI, mean fluorescence intensity; MOI, multiplicity of infection.

Credit

Genes & Diseases

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