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METHODS AND COMPOSITIONS FOR TARGETED GENETIC MODIFICATION USING PAIRED GUIDE RNAs.

机译:使用配对指导RNA进行有针对性的遗传修饰的方法和成分。

摘要

Compositions and methods are provided for creating and promoting biallelic targeted modifications to genomes within cells and for producing non-human animals comprising the modified genomes. Also provided are compositions and methods for modifying a genome within a cell that is heterozygous for an allele to become homozygous for that allele. The methods make use of Cas proteins and two or more guide RNAs that target different locations within the same genomic target locus. Also provided are methods of identifying cells with modified genomes.
机译:提供了组合物和方法,用于产生和促进对细胞内基因组的双等位基因靶向修饰以及用于产生包含修饰的基因组的非人类动物。还提供了用于修饰细胞中的基因组的组合物和方法,所述细胞对于等位基因是杂合的,对于所述等位基因是纯合的。所述方法利用Cas蛋白和靶向相同基因组靶基因座内不同位置的两个或更多个指导RNA。还提供了鉴定具有修饰的基因组的细胞的方法。

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