Figure 1 | Scientific Reports

Figure 1

From: AAV2-mediated gene therapy for Bietti crystalline dystrophy provides functional CYP4V2 in multiple relevant cell models

Figure 1

CYP4V2 expression in human RPE and plasmids CYP4V2-encoding plasmids. (A) CYP4V2 is highly expressed in human RPE cells (TPM: transcripts per million) and ranks 4th among other CYP family genes, as determined by RNA-seq. (B) Schematic diagrams of plasmids encoding wild-type or codon-optimized CYP4V2 transgenes driven by a CAG (CMV-enhanced chicken beta-actin) promoter. (C and D) Strong CYP4V2 expression was observed in HEK293 cells transfected with p.coCYP4V2 compared to those transfected with p.wtCYP4V2, assessed by western blot and immunocytochemistry analysis, respectively. (E) CYP enzyme activity was measured in HEK293 cells transfected with p.eGFP, p.Exon7Del, p.992A > C p.CYP4X1, p.CYP1B1, p.wtCYP4V2 and p.coCYP4V2. The latter four plasmid transfections were exposed to a dose response experiment with HET0016, a selective inhibitor of CYP4V2. Data are presented as the mean ± SEM (n = 6). ****p < 0.00001. ns, not significant.

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