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  1. ...(Supplemental Fig. S6), consistent with observations from other Cas9-mediated editing studies (Haapaniemi et al. 2018; Enache et al. 2020). Despite these changes, cell morphology and colony formation efficiency were unaffected, underscoring V2.1G's capability to generate robust multigene-targeting clones...
  2. ...expression of the holoenzyme from a single adeno-associated viral vector for in vivo editing applications. Delivery of St1Cas9 to the neonatal liver efficiently rewired metabolic pathways, leading to phenotypic rescue in a mouse model of hereditary tyrosinemia. These robust enzymes expand and complement...
  3. ..., Kim D, Cho SW, Kim J, Kim JS. 2014. Highly efficient RNA-guided editing in human cells via delivery of purified Cas9 ribonucleoproteins. Genome Res 24: 1012–1019. ↵Kim D, Bae S, Park J, Kim E, Kim S, Yu HR, Hwang J, Kim JI, Kim JS. 2015. Di-seq: -wide profiling of CRISPR-Cas9 off-target effects...
  4. ...– 10625. ChangN, SunC, Gao L, ZhuD, Xu X, Zhu X, Xiong JW, Xi JJ. 2013. Genome editing with RNA-guided Cas9 nuclease in zebrafish embryos. Cell Res 23: 465–472. Cho SW, Kim S, Kim JM, Kim JS. 2013a. Targeted engineering in human cells with the Cas9 RNA-guided endonuclease.Nat Biotechnol31: 230–232. Cho SW...
  5. ...meganucleases is transforming editing and functional genomics. CRISPR/Cas9 was developed such that targeted genomic lesions could be introduced in vivo with unprecedented ease. In the presence of homology donors, these lesions facilitate high-efficiency precise editing (PGE) via homology-directed repair (HDR...
  6. ...to efficiently capture and dissect the on-target sequence content in the order of kilobases. We resolved complex on-target genomic rearrangements with varying biological consequences. By introducing a powerful approach, this study contributes to a more reliable validation of CRISPR-Cas9-induced deletions...
  7. ..., Li Y, Fine EJ, Wu X, Shalem O, et al. 2013. DNA targeting specificity of RNA-guided Cas9 nucleases. Nat Biotechnol 31: 827–832. HwangWY, FuY, ReyonD,MaederML, Tsai SQ, Sander JD, Peterson RT, Yeh JR, Joung JK. 2013. Efficient editing in zebrafish using a CRISPR-Cas system. Nat Biotechnol 31: 227...
  8. ...J, Kim JS. 2014. Highly efficient RNA-guided editing in human cells via delivery of purified Cas9 ribonucleoproteins. Genome Res (this issue). doi: 10.1101/gr.171322.113. Li D, Qiu Z, Shao Y, Chen Y, Guan Y, Liu M, Li Y, Gao N, Wang L, Lu X, et al. 2013a. Heritable gene targeting in themouse and rat...
  9. ...efficient RNA-guided editing in human cells via delivery of purified Cas9 ribonucleoproteins. Genome Res 24: 1012–1019. doi:10.1101/gr.171322.113 ↵Kolonin MG, Saha PK, Chan L, Pasqualini R, Arap W. 2004. Reversal of obesity by targeted ablation of adipose tissue. Nat Med 10: 625–632. doi:10.1038/nm1048...
  10. ...proteins, TALEs/TALENs, and the CRISPR/Cas9 system as nucleases for genome editing, transcription factors for epigenome editing, and other emerging applications. We also present current and potential future applications of these tools, as well as their current limitations and areas for future advances...
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