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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)
In article number https://doi.org/10.1002/advs.201903432, 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 e...
| Autores principales: | , , , , , , , , , , , , , , , , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
John Wiley and Sons Inc.
2020
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7237839/ http://dx.doi.org/10.1002/advs.202070054 |
| _version_ | 1783536406859087872 |
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| author | 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 |
| author_facet | 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 |
| author_sort | Chou, Shih‐Jie |
| collection | PubMed |
| description | In article number https://doi.org/10.1002/advs.201903432, 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. [Image: see text] |
| format | Online Article Text |
| id | pubmed-7237839 |
| institution | National Center for Biotechnology Information |
| language | English |
| publishDate | 2020 |
| publisher | John Wiley and Sons Inc. |
| record_format | MEDLINE/PubMed |
| spelling | pubmed-72378392020-05-21 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) 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 Adv Sci (Weinh) Frontispiece In article number https://doi.org/10.1002/advs.201903432, 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. [Image: see text] John Wiley and Sons Inc. 2020-05-20 /pmc/articles/PMC7237839/ http://dx.doi.org/10.1002/advs.202070054 Text en © 2020 WILEY‐VCH Verlag GmbH & Co. KGaA, Weinheim This is an open access article under the terms of the http://creativecommons.org/licenses/by-nc/4.0/ License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited and is not used for commercial purposes. |
| spellingShingle | Frontispiece 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 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) |
| 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) |
| title_full | 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) |
| title_fullStr | 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) |
| title_full_unstemmed | 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) |
| title_short | 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) |
| title_sort | 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) |
| topic | Frontispiece |
| url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7237839/ http://dx.doi.org/10.1002/advs.202070054 |
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