The invention provides xenonucleic acids and synthetic chimeric xenonucleic acid guide RNA; s(XNA-gRNA) for enhancing crispr mediated genome editing efficiency. The invention also provides methods and compositions for inducing CRISPR/Cas-based gene editing/regulation (e.g., genome editing or gene expression) of a target nucleic acid (e.g., target DNA or target RNA) in a cell. The methods include using single guide RNAs (sgRNAs) that have been chemically modified with xeno nucleic acids which enhance gene regulation of the target nucleic acid in a primary cell for use in ex vivo therapy or in a cell in a subject for use in in vivo therapy. Additionally, provided herein are methods for preventing or treating a genetic disease in a subject by administering a sufficient amount of a sgRNA that has been chemically modified with xeno nucleic acids to correct a mutation in a target gene associated with the genetic disease.
该发明提供了xenonucleic acids和合成嵌合xenonucleic acid guide RNA;s(XNA-gRNA),用于增强cri
SPr介导的
基因组编辑效率。该发明还提供了诱导CRI
SPR/Cas
基因编辑/调控(例如
基因组编辑或
基因表达)靶向核酸(例如靶向DNA或靶向RNA)在细胞中的方法和组合物。这些方法包括使用已经经过
化学修饰的xeno核酸的单导RNA(sgRNAs),这些核酸增强了原代细胞中靶向核酸的
基因调控,用于体外治疗或用于体内治疗中的受体细胞。此外,本文提供了通过向受体注射已经经过
化学修饰的xeno核酸的足够数量的sgRNA来预防或治疗受体的遗传疾病的方法,以纠正与该遗传疾病相关的靶
基因的突变。