Efficacy and dynamics of self-targeting CRISPR/Cas constructs for gene editing in the retina.
Li Fan, Sandy S. C. Hung, Mohd Khairul Nizam Mohd Khalid, Jiang-Hui Wang, Vicki Chrysostomou, Vickie H. Y. Wong, Vikrant Singh, Kristof Wing, Leilei Tu, James A. Bender, Alice Pébay, Anna Elizabeth King, Anthony L. Cook, Raymond C.B. Wong, Bang Viet Bui, , Guei‐Sheung Liu
This study develops a self-destructing CRISPR/Cas system for retinal gene editing, showing reduced SpCas9 mRNA and improved safety without compromising on-target efficiency.
Human Gene Therapy · Aug 2, 2019Peer-reviewedGenetics