![]() ![]() Open angle glaucoma (OAG) is a complex ocular disorder affecting approximately 79.6 million individuals worldwide resulting in progressive loss of retinal ganglion cells, axonal damage within the optic nerve, and ultimately, blindness 1. Longitudinal multimodal imaging, electrophysiology, and post-mortem histology revealed the therapy was well tolerated at low and medium doses, with no major adverse effects to anterior chamber health, offering a promising alternative to current treatment strategies leading to clinically relevant reductions in intraocular pressure without the need for adherence to a daily treatment regimen. Crucially, therapy could be temporarily halted through off-type riboswitch activation, reverting intraocular pressure to normal. This study demonstrated a dose dependent reduction in intraocular pressure in normotensive Brown Norway rats maintained over 12-months. ![]() ![]() Herein, we evaluate the safety and efficacy of a recombinant adeno-associated viral vector-mediated gene therapy aimed at permanently lowering intraocular pressure through de novo biosynthesis of prostaglandin F2α within the anterior chamber. ![]() Prostaglandin analogs are first-line treatments for open angle glaucoma and while effective at lowering intraocular pressure, they are undermined by patient non-compliance, causing atrophy of the optic nerve and severe visual impairment. ![]()
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