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Gene Therapy: Dual Supramolecular Nanoparticle Vectors Enable CRISPR/Cas9‐Mediated Knockin of Retinoschisin 1 Gene—A Potential Nonviral Therapeutic Solution for X‐Linked Juvenile Retinoschisis (Adv. Sci. 10/2020)
Advanced Science ( IF 15.804 ) Pub Date : 2020-05-20 , DOI: 10.1002/advs.202070054
Shih‐Jie Chou; Peng Yang; Qian Ban; Yi‐Ping Yang; Mong‐Lien Wang; Chian‐Shiu Chien; Shih‐Jen Chen; Na Sun; Yazhen Zhu; Hongtao Liu; Wenqiao Hui; Tai‐Chi Lin; Fang Wang; Ryan Yue Zhang; Viet Q. Nguyen; Wenfei Liu; Mengxiang Chen; Steve J. Jonas; Paul S. Weiss; Hsian‐Rong Tseng; Shih‐Hwa Chiou

In article number 1903432, Shih‐Hwa Chiou, Hsian‐Rong Tseng, Paul S. Weiss, and co‐workers introduce an in vivo CRISPR/Cas9‐mediated knockin approach using two supramolecular nanoparticle (SMNP) vectors. By intravitreally injecting the two SMNP vectors into the mouse eyes, the RS1/GFP gene is knocked into the Rosa26 site in mice retinas, offering a revolutionarily curative therapeutic solution for X‐linked juvenile retinoschisis.
更新日期:2020-05-20

 

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