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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)
Advanced Science ( IF 14.3 ) Pub Date : 2020-05-20 , DOI: 10.1002/advs.202070054
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, Wenqiao Hui, Tai‐Chi Lin, Fang Wang, Ryan Yue Zhang, Viet Q. Nguyen, Wenfei Liu, Mengxiang Chen, Steve J. Jonas, Paul S. Weiss, Hsian‐Rong Tseng, Shih‐Hwa Chiou

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 revolutionarily curative therapeutic solution for X‐linked juvenile retinoschisis.
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中文翻译:

基因治疗:双超分子纳米载体可实现CRISPR / Cas9介导的视黄酸镰刀菌素1基因的敲除-一种X连锁的青少年视网膜视胶症的潜在非病毒治疗方案(Adv。Sci。10/2020)

Shih-Hwa Chiou,Hsian-Rong Tseng,Paul S.Weiss和同事在文章1903432中介绍了使用两种超分子纳米(SMNP)载体的体内CRISPR / Cas9介导的敲入方法。通过将两种SMNP载体玻璃体内注射到小鼠眼中,RS1 / GFP基因敲入小鼠视网膜中的Rosa26位点,为X连锁的青少年视网膜裂变提供了革命性的治疗方案。
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更新日期:2020-05-20
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