Long-read RNA-Seq analysis of the full-length F8 transcript in iPSCs
We repaired the variant in iPSCs derived from a patient with moderate hemophilia A.  To confirm the restoration of the full-length F8 transcript in the gene-corrected iPSCs, we performed long-read RNA-Seq analysis in iPSCs with the EF1a promoter. Overall design: Full-length F8 transcript in healthy donor-derived iPSCs or variant-corrected or uncorrected patient-derived iPSCs