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58. Gaussin A, Modlich U, Bauche C, et al: CTF/NF1 transcription factors act as potent 62. DeKelver RC, Choi VM, Moehle EA, et al: Functional genomics, proteomics, and regu-
genetic insulators for integrating gene transfer vectors. Gene Ther 19(1):15–24, 2012. latory DNA analysis in isogenic settings using zinc finger nuclease-driven transgenesis
59. Heckl D, Schwarzer A, Haemmerle R, et al: Lentiviral vector induced insertional haplo- into a safe harbor locus in the human genome. Genome Res 20(8):1133–1142, 2010.
insufficiency of Ebf1 causes murine leukemia. Mol Ther 20(6):1187–1195, 2012. 63. Urnov FD, Miller JC, Lee YL, et al: Highly efficient endogenous human gene correction
60. Fung H, Weinstock DM: Repair at single targeted DNA double-strand breaks in using designed zinc-finger nucleases. Nature 435(7042):646–651, 2005.
pluripotent and differentiated human cells. PLoS One 6(5):e20514, 2011. 64. Reyon D, Tsai SQ, Khayter C, et al: FLASH assembly of TALENs for high-throughput
61. Smith JR, Maguire S, Davis LA, et al: Robust, persistent transgene expression in genome editing. Nat Biotechnol 30(5):460–465, 2012.
human embryonic stem cells is achieved with AAVS1-targeted integration. Stem Cells 65. Sander JD, Joung JK: CRISPR-Cas systems for editing, regulating and targeting
26(2):496–504, 2011. genomes. Nat Biotechnol 32(4):347–355, 2014.

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