引导RNA
清脆的
基因组编辑
Cas9
核糖核蛋白
核糖核酸
生物
计算生物学
基因组
DNA
核酸内切酶
RNA编辑
遗传学
基因
作者
Ayal Hendel,Rasmus O. Bak,Joseph T. Clark,Andrew Kennedy,Daniel E. Ryan,Susmita Roy,Israel Steinfeld,Benjamin D Lunstad,Robert Kaiser,Alec B. Wilkens,Rosa Bacchetta,Anya Tsalenko,Douglas J. Dellinger,Laurakay Bruhn,Matthew H. Porteus
摘要
Improved efficiency of CRISPR/Cas editing with chemically modified synthetic sgRNAs. CRISPR-Cas-mediated genome editing relies on guide RNAs that direct site-specific DNA cleavage facilitated by the Cas endonuclease. Here we report that chemical alterations to synthesized single guide RNAs (sgRNAs) enhance genome editing efficiency in human primary T cells and CD34+ hematopoietic stem and progenitor cells. Co-delivering chemically modified sgRNAs with Cas9 mRNA or protein is an efficient RNA- or ribonucleoprotein (RNP)-based delivery method for the CRISPR-Cas system, without the toxicity associated with DNA delivery. This approach is a simple and effective way to streamline the development of genome editing with the potential to accelerate a wide array of biotechnological and therapeutic applications of the CRISPR-Cas technology.
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