Article
PRPF8 Mutation-Induced Defects in Human iPSC-Derived RPE Are Rescued by Adenine Base Editing.
Investigative ophthalmology & visual science - 5 Jan 2026
Sun Xihao, Liang Yuan, Liang Yuqin, Li Wenwei, Chen Hang, Ding Chengcheng, Duan Chunwen, Zhou Yalan, Zhang Ruting, Xu Huizhuo, Chen Jiansu
Abstract excerpt
Purpose: The pathological effects of pre-mRNA processing factor 8 (PRPF8) mutations on the retinal pigment epithelium (RPE) are not fully understood. We aimed to identify disease-specific cellular and molecular phenotypes in PRPF8 retinitis pigmentosa (RP) patient-derived induced pluripotent stem cell (iPSC)-RPE and to test whether adenine base editing (ABE), which corrects the PRPF8 mutation in iPSCs, can...
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