| Autor: |
Chern, Kristina J., Nettesheim, Emily R., Reid, Christopher A., Li, Nathan W., Marcoe, Gavin J., Lipinski, Daniel M. |
| Zdroj: |
Communications Biology; 11/3/2022, Vol. 5 Issue 1, p1-9, 9p |
| Abstrakt: |
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. 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. This study demonstrated a dose dependent reduction in intraocular pressure in normotensive Brown Norway rats maintained over 12-months. Crucially, therapy could be temporarily halted through off-type riboswitch activation, reverting intraocular pressure to normal. 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.A well-tolerated rAAV vector-mediated gene therapy in normotensive Brown Norway rats is used to reduce intraocular pressure in a dose dependent manner, encouraging further evaluations on pre-clinical models of glaucoma. [ABSTRACT FROM AUTHOR] |
| Databáze: |
Complementary Index |
| Externí odkaz: |
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