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). Issue 10 (20th May 2020)

• Record Type: Journal Article
• Authors: Chou, Shih‐Jie; Yang, Peng; Ban, Qian; Yang, Yi‐Ping; Wang, Mong‐Lien; Chien, Chian‐Shiu; Chen, Shih‐Jen; Sun, Na; Zhu, Yazhen; Liu, Hongtao; Hui, Wenqiao; Lin, Tai‐Chi; Wang, Fang; Zhang, Ryan Yue; Nguyen, Viet Q.; Liu, Wenfei; Chen, Mengxiang; Jonas, Steve J.; Weiss, Paul S.; Tseng, Hsian‐Rong; Chiou, Shih‐Hwa
• Is Part Of: Advanced science. Volume 7:Issue 10(2020)
• Page Start: n/a
• Page End: n/a
• Publication Date: 2020-05-20
• Subjects: codelivery -- CRISPR/Cas9 -- gene therapy -- retina -- supramolecular nanoparticles -- X‐linked juvenile retinoschisis; Science -- Periodicals; 505
• DOI: 10.1002/advs.202070054
• Languages: English
• ISSNs: 2198-3844

Abstract

Abstract : 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.