Dual Supramolecular Nanoparticle Vectors Enable CRISPR/Cas9‐Mediated Knockin of Retinoschisin 1 Gene...
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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Author / Creator
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 and Chiou, Shih‐Hwa
Publisher
Germany: John Wiley & Sons, Inc
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English
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Germany: John Wiley & Sons, Inc
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Contents
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...
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Full title
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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TN_cdi_doaj_primary_oai_doaj_org_article_73b313974d854f8cbb56eec82f8ba8d4
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_73b313974d854f8cbb56eec82f8ba8d4
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ISSN
2198-3844
E-ISSN
2198-3844
DOI
10.1002/advs.201903432
