![]() 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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