Figure 2 | Scientific Reports

Figure 2

From: The pathogenesis linked to coenzyme Q10 insufficiency in iPSC-derived neurons from patients with multiple-system atrophy

Figure 2

Site-specific gene correction. (A) Schematic overview for gene repair using CRISPR/Cas9 and piggyBac transposon methodology. Small arrows indicate primers for PCR and Sanger sequencing base screening to confirm the selected clones. PBx, excision-only piggyBac transposase; ITR, inverted terminal repeat; TK, thymidine kinase; Exon 7*, mutated exon 7; Exon 7**, donor exon 7. (B) Sanger DNA sequencing confirming of the gene edition. An iPSC clone (MSA_A26E-37) was identified as homozygous targeted. (C) Foot print analysis by sequencing. Sequences in the cassette-free repair clones indicated the exact repair after piggyBac excision. TTAA target sites are boxed. (D) Karyotype analysis. Four correctly repaired clones were confirmed normal karyotypes.

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