PMC:5985359 / 35286-36243 JSONTXT

{"project":"2_test","denotations":[{"id":"29526280-26325627-2044801","span":{"begin":791,"end":793},"obj":"26325627"}],"text":"To assess the likely accessibility of ASOs to the corneal endothelium, C57B16 mice were injected intravitreally with varying concentrations (0.025, 0.01, and 0.005 mg) of Cy-3-labeled 2′Ome-PS-(CAG)7. Confocal fluorescence microscopy revealed that the ASO was present in corneal endothelium, keratocytes, and stroma, specifically accumulating in both the nuclear and perinuclear region of both the endothelial and stromal cells. This localization increased in a dose-dependent manner, showing stronger accumulation in cell layers at 48 hr post-dosing (Figure S6). ASOs with identical chemistry, following intraocular injection, have previously been shown in vivo to display a similar peri-nuclear and nuclear localization in other cell types in conjunction with effective molecular activity.38 These data highlight the potential for the (CAG)7 ASO to target CECs in vivo, an essential prerequisite for the effective delivery of an ASO-mediated FECD therapy."}