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…·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.