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Publication: Toualbi et al., 2023 - Gene Augmentation of CHM Using Non-Viral Episomal Vectors in Models of Choroideremia
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Figure 1

Generation of pS/MAR-CHM vectors. (A) pS/MAR-CHM vectors were generated by inserting the human CHM coding sequence (CDS) into the S/MAR plasmid backbone with 1 of 5 promoters: CMV (V1), CAG (V2), hPGK (V3), EF1α (V4), or CHMp (V5). The plasmids also contained a GFP sequence. (B) All 5 vectors produced GFP expression in transfected HEK-293 cells, which was observed at varying levels at 48 h post-transfection. (C) REP1 protein expression was examined by Western blot in the non-transfected (NT) and transfected HEK-293 cells, with the CMV (V1) and CAG (V2) promoter versions driving the highest expression levels.

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