Fig. 1: Construct schematic and confirmation of in vitro activity and in vivo tropism.
From: Prostaglandin-based rAAV-mediated glaucoma gene therapy in Brown Norway rats
A diagrammatical representation of the CCPP transgene driven by a CMV enhancer and smCBA promotor while containing two tetracycline off-type riboswitches (TC40 and TC45), as well as human codon optimized COX2 and PTGFR separated by a P2A cleavage signal (a). A PGF2ɑ ELISA confirmed significantly higher concentrations of PGF2ɑ in cell culture media after transfection with CCPP transgene than media collected from sham transfected cells (b) (unpaired T-test, p = 0.0056, N = 2 (sham transfected) N = 3 (CCPP transfected), error bars = SD). Brightfield and fluorescent imaging of rAAV2/2[MAX]-smCBA-mCherry eyes 8-weeks post injection confirm vector tropism within the iris, corneal endothelium, and ciliary body (c, d). Scale bar = 150 µm, Magnification = 20×.
