Multiple copies of a linear donor fragment released in situ from a vector improve the efficiency of zinc-finger nuclease-mediated genome editing

Gene Therapy

Volumn 21, Issue 3, 2014, Pages 282-288

  Zhang, W ^a   Wang, D ^a   Liu, S ^a   Zheng, X ^a   Ji, H ^a   Xia, H ^a   Mao, Q ^b  

^a SHAANXI NORMAL UNIVERSITY   (China)

^b NORTHWESTERN UNIVERSITY   (United States)

Author keywords

adenoviral vector; genome editing; homologous recombination; site specific integration; zinc finger nuclease

Indexed keywords

ADENOVIRUS VECTOR; DNA FRAGMENT; LENTIVIRUS VECTOR; PLASMID VECTOR; ZINC FINGER NUCLEASE;

ARTICLE; CELL STRAIN HEK293; DNA CLEAVAGE; DNA FLANKING REGION; EMBRYO; EXPRESSION VECTOR; GENE DOSAGE; GENE TARGETING; GENE THERAPY; HOMOLOGOUS RECOMBINATION; HUMAN; HUMAN CELL; HUMAN CELL CULTURE; PRIORITY JOURNAL; PROTEIN EXPRESSION; TANDEM REPEAT;

ADENOVIRIDAE; CELL LINE, TUMOR; DEOXYRIBONUCLEASE I; GENE TARGETING; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; GENOME, HUMAN; HEK293 CELLS; HOMOLOGOUS RECOMBINATION; HUMANS; ZINC FINGERS;

EID: 84896730576     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2013.83     Document Type: Article

References (39)

1. Capecchi MR. Altering the genome by homologous recombination. Science 1989; 244: 1288-1292. (Pubitemid 19170497)
2. Koller BH, Smithies O. Altering genes in animals by gene targeting. Annu Rev Immunol 1992; 10: 705-730.
3. Bibikova M, Beumer K, Trautman JK, Carroll D. Enhancing gene targeting with designed zinc finger nucleases. Science 2003; 300: 764. (Pubitemid 36532104)
4. Brenneman M, Gimble FS, Wilson JH. Stimulation of intrachromosomal homologous recombination in human cells by electroporation with site-specific endonucleases. Proc Natl Acad Sci USA 1996; 93: 3608-3612. (Pubitemid 26125668)
5. Choulika A, Perrin A, Dujon B, Nicolas JF. Induction of homologous recombination in mammalian chromosomes by using the I-SceI system of Saccharomyces cerevisiae. Mol Cell Biol 1995; 15: 1968-1973.
6. Porteus MH, Baltimore D. Chimeric nucleases stimulate gene targeting in human cells. Science 2003; 300: 763. (Pubitemid 36532103)
7. Rouet P, Smih F, Jasin M. Expression of a site-specific endonuclease stimulates homologous recombination in mammalian cells. Proc Natl Acad Sci USA 1994; 91: 6064-6068. (Pubitemid 24188283)
8. Sargent RG, Brenneman MA, Wilson JH. Repair of site-specific double-strand breaks in a mammalian chromosome by homologous and illegitimate recombination. Mol Cell Biol 1997; 17: 267-277. (Pubitemid 26425616)
9. Smih F, Rouet P, Romanienko PJ, Jasin M. Double-strand breaks at the target locus stimulate gene targeting in embryonic stem cells. Nucleic Acids Res 1995; 23: 5012-5019. (Pubitemid 26012483)
10. Goncz KK, Prokopishyn NL, Chow BL, Davis BR, Gruenert DC. Application of SFHR to gene therapy of monogenic disorders. Gene Ther 2002; 9: 691-694. (Pubitemid 34591467)
11. Orr-Weaver TL, Szostak JW, Rothstein RJ. Yeast transformation: a model system for the study of recombination. Proc Natl Acad Sci USA 1981; 78: 6354-6358. (Pubitemid 12179710)
12. Bedell VM, Wang Y, Campbell JM, Poshusta TL, Starker CG, Krug 2nd RG et al. In vivo genome editing using a high-efficiency TALEN system. Nature 2012; 491: 114-118.
13. Boch J, Scholze H, Schornack S, Landgraf A, Hahn S, Kay S et al. Breaking the code of DNA binding specificity of TAL-type III effectors. Science 2009; 326: 1509-1512.
14. Moscou MJ, Bogdanove AJ. A simple cipher governs DNA recognition by TAL effectors. Science 2009; 326: 1501.
15. Wood AJ, Lo TW, Zeitler B, Pickle CS, Ralston EJ, Lee AH et al. Targeted genome editing across species using ZFNs and TALENs. Science 2011; 333: 307.
16. Cho SW, Kim S, Kim JM, Kim JS. Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease. Nat Biotechnol 2013; 31: 230-232.
17. Cong L, Ran FA, Cox D, Lin S, Barretto R, Habib N et al. Multiplex genome engineering using CRISPR/Cas systems. Science 2013; 339: 819-823.
18. Jinek M, Chylinski K, Fonfara I, Hauer M, Doudna JA, Charpentier E. A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity. Science 2012; 337: 816-821.
19. Mali P, Yang L, Esvelt KM, Aach J, Guell M, DiCarlo JE et al. RNA-guided human genome engineering via Cas9. Science 2013; 339: 823-826.
20. Kunzelmann K, Legendre JY, Knoell DL, Escobar LC, Xu Z, Gruenert DC. Gene targeting of CFTR DNA in CF epithelial cells. Gene Ther 1996; 3: 859-867. (Pubitemid 26348339)
21. Gong WJ, Golic KG. Ends-out, or replacement, gene targeting in Drosophila. Proc Natl Acad Sci USA 2003; 100: 2556-2561. (Pubitemid 36297537)
22. de Piédoue G, Maurisse R, Kuzniak I, Lopez B, Perrin A, Nègre O et al. Improving gene replacement by intracellular formation of linear homologous DNA. J Gene Med 2005; 7: 649-656.
23. Zhang W, Zheng X, Wang Y, Mao Q, Xia H. Establishment of a cell line carrying single copy of an exogenous mutant reporter gene for assaying the biological activity of ZFNs. J Biotechnol 2012; 162: 191-196.
24. Doyon Y, Vo TD, Mendel MC, Greenberg SG, Wang J, Xia DF et al. Enhancing zinc-finger-nuclease activity with improved obligate heterodimeric architectures. Nat Methods 2011; 8: 74-79.
25. Orlando SJ, Santiago Y, DeKelver RC, Freyvert Y, Boydston EA, Moehle EA et al. Zinc-finger nuclease-driven targeted integration into mammalian genomes using donors with limited chromosomal homology. Nucleic Acids Res 2010; 38: e152.
26. Deng C, Capecchi MR. Reexamination of gene targeting frequency as a function of the extent of homology between the targeting vector and the target locus. Mol Cell Biol 1992; 12: 3365-3371.
27. Hasty P, Rivera-Pérez J, Bradley A. The length of homology required for gene targeting in embryonic stem cells. Mol Cell Biol 1991; 11: 5586-5591. (Pubitemid 21895325)
28. te Riele H, Maandag ER, Berns A. Highly efficient gene targeting in embryonic stem cells through homologous recombination with isogenic DNA constructs. Proc Natl Acad Sci USA 1992; 89: 5128-5132.
29. Thomas KR, Capecchi MR. Site-directed mutagenesis by gene targeting in mouse embryoderived stem cells. Mol Cell Biol 1987; 51: 503-512.
30. Shulman MJ, Nissen L, Collins C. Homologous recombination in hybridoma cells: dependence on time and fragment length. Mol Cell Biol 1990; 10: 4466-4472.
31. Cristea S, Freyvert Y, Santiago Y, Holmes MC, Urnov FD, Gregory PD et al. In vivo cleavage of transgene donors promotes nuclease-mediated targeted integration. Biotechnol Bioeng 2012; 110: 871-880.
32. Connelly JP, Barker JC, Pruett-Miller S, Porteus MH. Gene correction by homologous recombination with zinc finger nucleases in primary cells from a mouse model of a generic recessive genetic disease. Mol Ther 2010; 18: 1103-1110.
33. Li H, Haurigot V, Doyon Y, Li T, Wong SY, Bhagwat AS et al. In vivo genome editing restores haemostasis in a mouse model of haemophilia. Nature 2011; 475: 217-221.
34. Soldner F, Laganiè re J, Cheng AW, Hockemeyer D, Gao Q, Alagappan R et al. Generation of isogenic pluripotent stem cells differing exclusively at two early onset Parkinson point mutations. Cell 2011; 146: 318-331.
35. Mohammadi ES, Ketner EA, Johns DC, Ketner G. Expression of the adenovirus E4 34k oncoprotein inhibits repair of double strand breaks in the cellular genome of a 293-based inducible cell line. Nucleic Acids Res 2004; 32: 2652-2659. (Pubitemid 38854819)
36. Hockemeyer D, Soldner F, Beard C, Gao Q, Mitalipova M, DeKelver RC et al. Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases. Nat Biotechnol 2009; 27: 851-857.
37. Zhang W, Guo Y, Zhang C, Ji H, Meng W, Wang D et al. Rescue the failed half-ZFN by a sensitive mammalian cell-based luciferase reporter system. PLoS One 2012; 7: e45169.
38. Perez-Pinera P, Ousterout DG, Brown MT, Gersbach CA. Gene targeting to the ROSA26 locus directed by engineered zinc finger nucleases. Nucleic Acids Res 2012; 40: 3741-3752.
39. Anderson RD, Haskell RE, Xia H, Roessler BJ, Davidson BL. A simple method for the rapid generation of recombinant adenovirus vectors. Gene Ther 2000; 7: 1034-1038. (Pubitemid 30398352)