Figure 4.
Figure 4. Functional analysis of myeloid cells generated from homozygous Δ734-748 NCF1 p47-CGD iPSCs corrected by exon 2 replacement. (A) p47phox expression in myeloid cells differentiated from iPSCs derived from the homozygous Δ734-748 NCF1 p47-CGD subject, including the uncorrected line, P47-05 (first panel on the left), 7 exon 2 replacement (GT ins) clones, P47-05-C1 to C7 (middle 7 panels), and the healthy control iPSCs (last panel on the right). (B) Oxidase function in corresponding clones is shown by DHR assay. The DHR assay, gating for MFI assessment, and donor target assessment are as described in the Figure 2D legend.

Functional analysis of myeloid cells generated from homozygous Δ734-748 NCF1 p47-CGD iPSCs corrected by exon 2 replacement. (A) p47phox expression in myeloid cells differentiated from iPSCs derived from the homozygous Δ734-748 NCF1 p47-CGD subject, including the uncorrected line, P47-05 (first panel on the left), 7 exon 2 replacement (GT ins) clones, P47-05-C1 to C7 (middle 7 panels), and the healthy control iPSCs (last panel on the right). (B) Oxidase function in corresponding clones is shown by DHR assay. The DHR assay, gating for MFI assessment, and donor target assessment are as described in the Figure 2D legend.

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