Figure 3.
Phenotypic Rescue of the Expression of FVIII Gene from the Corrected iPSC Clones
(A) RT-PCR analysis to detect the expression of FVIII and OCT4 in undifferentiated, corrected iPSC clones. GAPDH expression was used for normalization.
(B) Chromatograms showing the sequences of amplified DNA bands from each clone (related to A).
(C) Results of the qPCR analysis showing the FVIII expression levels in undifferentiated patient iPSC clones and corrected clones. GAPDH expression was used for normalization. Data are means ± SEM of three independent experiments. ND, not detected.
(D) The FVIII activity was determined after 20-fold concentration in supernatants obtained from either patient or corrected clones. Data indicate activity detected per 1 × 106 iPSCs. Data are means ± SEM of three independent experiments.