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CAS EXPRESSING MOUSE EMBRYONIC STEM CELLS AND MOUSE AND THEIR APPLICATIONS
CAS EXPRESSING MOUSE EMBRYONIC STEM CELLS AND MOUSE AND THEIR APPLICATIONS
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机译:CAS表达小鼠胚胎干细胞和小鼠及其应用
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摘要
Methods and compositions are provided herein for assessing CRISPR/Cas-mediated non-homologous end joining (NHEJ) activity and/or CRISPR/Cas-induced recombination of a target genomic locus with an exogenous donor nucleic acid in vivo and ex vivo. The methods and compositions employ cells and non-human animals comprising a Cas expression cassette such as a genomically integrated Cas expression cassette so that the Cas protein can be constitutively available or available in a tissue-specific or temporal-specific manner. Methods and compositions are also provided for making and using these non-human animals, including use of these non-human animals to assess CRISPR/Cas activity in vivo via adeno-associated virus (AAV)-mediated delivery of guide RNAs to the non-human animals.
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