Gene Therapy: Dual Supramolecular Nanoparticle Vectors Enable CRISPR/Cas9‐Mediated Knockin of Retino...
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)
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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
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Weinheim: John Wiley & Sons, Inc
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English
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Weinheim: John Wiley & Sons, Inc
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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...
Alternative Titles
Full title
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)
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TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_7237839
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_7237839
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ISSN
2198-3844
E-ISSN
2198-3844
DOI
10.1002/advs.202070054
