Identification a Compact Promoter using a New Promoter Selection Strategy and Engineering Hybrid Pol II/III Enable Efficient Genome Editing in Human Retinal Ganglion Cells
Pingwu Zhang, Steven H. Zhang, Yen-Yu Chang, Sean Li, Laura Fan, Weifeng Li, Yukan Duan, Jie Cheng, Casey J. Keuthan, Cynthia A. Berlinicke, Derek S. Welsbie, Donald J. Zack
The paper identifies a compact Pol II promoter and hybrid Pol II/III promoters to enable efficient single-AAV delivery of SpCas9 for genome editing in human retinal ganglion cells.
bioRxiv · Jan 28, 2026 · PreprintMolecular Biology