RHO1-2 meganuclease gene editing targets human P23H rhodopsin-induced retinitis pigmentosa to rejuvenate rods and maintain cones.
RHO1-2 巨核酸酶基因编辑靶向人类 P23H 视紫红质诱导的视网膜色素变性,以恢复视杆细胞活力并维持视锥细胞功能。
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doi:10.1101/2025.09.25.678625
Jalligampala Archana, Young Jacob M, Feist Jack, Wang Wei, Barone Francesca, Alston David C, Fransen James W, Jaikumar Gita, Kamboj Kautuk, Mooreman Caitlin, Nash Stephen, Noel Jennifer M, Pangeni Gobinda, Prestigiacomo Joseph C, Sahu Bhubanananda, Turner Caitlin, Kaplan Henry J, Green Jonathan A, Wells Kevin D, Bartsevich Victor V, Chatterton Jon E, Davis Mara, Evans Kathryn S, Lape Janel, Lewis Whitney C, van de Beek Rebecca, Viles Kristi D, Jantz Derek, Gregg Ronald G, Smith Jeff, McCall Maureen A