Search-and-replace genome editing without double-strand breaks or donor DNA
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...…of a target gene in mammalian cells and organisms (Jinek et al., 2012; Cong et al., 2013;Mali et al., 2013; Mashiko et al., 2013; Kim et al., 2017; Anzalone et al., 2019), as compared with RNAi, which does not completely eradicate gene expression, and mRNA therapy, which transiently introduces…...
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154 citations
145 citations
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...corrected by targeting the four transition mutations, and in principle prime-editing could correct up to 89% of known genetic variants associated with human disease [9,10]....
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...Moreover, while Anzalone and coworkers observed no differences in viability and minimum changes in the cellular transcriptome of cell lines expressing inactivated RT, the clinical viability and safety of in vivo prime-editing remain to be tested [10]....
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...Prime-editors (PEs) are the latest addition to the CRISPR genome-engineering toolkit and represents a novel approach to expand the scope of donor-free precise DNA editing to not only all transition and transversion mutations, but small insertion and deletion mutations as well [10]....
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...The pegRNA contains not only (a) the sequence complimentary to the target sites that directs nCas9 to its target sequence, but also (b) an additional sequence spelling the desired sequence changes [10]....
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...1-fold improvement over PE1 Targets all transition/transversion mutations; small indels [10]...
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References
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