Dual Supramolecular Nanoparticle Vectors Enable CRISPR/Cas9‐Mediated Knockin of Retinoschisin 1 Gene—A Potential Nonviral Therapeutic Solution for X‐Linked Juvenile Retinoschisis

Dual Supramolecular Nanoparticle Vectors Enable CRISPR/Cas9‐Mediated Knockin of Retinoschisin 1 Gene—A Potential Nonviral Therapeutic Solution for X‐Linked Juvenile Retinoschisis

Abstract The homology‐independent targeted integration (HITI) strategy enables effective CRISPR/Cas9‐mediated knockin of therapeutic genes in nondividing cells in vivo, promising general therapeutic solutions for treating genetic diseases like X‐linked juvenile retinoschisis. Herein, supramolecular...

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Bibliographic Details
Main Authors: 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
Format: Article
Language:English
Published: Wiley 2020-05-01
Series:Advanced Science
Subjects:
codelivery
CRISPR/Cas9
gene therapy
retina
supramolecular nanoparticles
X‐linked juvenile retinoschisis
Online Access:https://doi.org/10.1002/advs.201903432

Internet

https://doi.org/10.1002/advs.201903432

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