RHO1-2 meganuclease gene editing targets human P23H rhodopsin-induced retinitis pigmentosa to rejuvenate rods and maintain cones
Archana Jalligampala, Jacob M. Young, Jack Feist, W. Wang, Francesca Barone, David C. Alston, James W. Fransen, Gita Jaikumar, Kautuk Kamboj, Caitlin Mooreman, Stephen Nash, Jennifer M. Noel, Gobinda Pangeni, Joseph Prestigiacomo, Bhubanananda Sahu, , Maureen A. McCall
This study demonstrates that RHO1-2 meganuclease gene editing can arrest rod degeneration and restore retinal function in a pig model of P23H rhodopsin-induced retinitis pigmentosa.
bioRxiv · Sep 25, 2025 · PreprintNeuroscience