Figure 1.

AAV9/JeT-GAN Construct Design for Expressing Human Gigaxonin

(A) Schematic diagram of the AAV/JeT-GAN gene transfer cassette using a JeT promoter, the full-length, codon-optimized human GAN cDNA, and the synthetic poly(A) tail (SpA). (B and C) Western blot and densitometry of gigaxonin expression from HEK293 cells transfected with the JeT-GAN plasmid or JeT-GFP control plasmid. (D and E) Western blot and densitometry of AAV9-permissive Lec2 cells treated with the indicated vectors at the indicated amounts per cell. (C and E) Values are mean ± SEM gigaxonin levels normalized to GAPDH or β-actin levels and relative to control (untransfected and GFP-treated) cells. Data were analyzed by one-way ANOVA with Bonerfonni’s post-hoc analysis.