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...

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Vydáno v:Adv Sci (Weinh)
Hlavní autoři: 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
Médium: Artigo
Jazyk:Inglês
Vydáno: John Wiley and Sons Inc. 2020
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On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC7237839/
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/advs.202070054
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