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

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; Paul S. Weiss; Hsian-Rong Tseng; Shih Hwa Chiou

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

doi.org/10.1002/advs.201903432

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

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..., Shih Hwa Chiou

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Advanced Science15.10

Volume: 7, Issue: 10

Published: Apr 16, 2020

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 nanoparticle (SMNP) vectors are used for codelivery of two DNA plasmids—CRISPR‐Cas9 genome‐editing system and a therapeutic gene, Retinoschisin 1...

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Paper Details

Title

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

DOI

doi.org/10.1002/advs.201903432

Published Date

Apr 16, 2020

Journal

Advanced Science

Volume

7

Issue

10

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