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.

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。