Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases

Nature Biotechnology

Volumn 27, Issue 9, 2009, Pages 851-857

  Hockemeyer, Dirk ^a   Soldner, Frank ^a   Beard, Caroline ^a   Gao, Qing ^a   Mitalipova, Maisam ^a   Dekelver, Russell C ^b   Katibah, George E ^b   Amora, Ranier ^b   Boydston, Elizabeth A ^b   Zeitler, Bryan ^b   Meng, Xiangdong ^b   Miller, Jeffrey C ^b   Zhang, Lei ^b   Rebar, Edward J ^b   Gregory, Philip D ^b   Urnov, Fyodor D ^b   Jaenisch, Rudolf ^a,c  

^a WHITEHEAD INSTITUTE FOR BIOMEDICAL RESEARCH   (United States)

^b SANGAMO BIOSCIENCES INC   (United States)

^c MASSACHUSETTS INSTITUTE OF TECHNOLOGY   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CELL TYPES; EFFICIENT METHOD; EXPRESSED GENES; GENE TARGETING; GENETIC MODIFICATIONS; HUMAN EMBRYONIC STEM CELLS; OVER-EXPRESSION; PLURIPOTENT; PLURIPOTENT STEM CELLS; SILENT GENES; TRANSGENE;

GENES; GENETIC ENGINEERING; ZINC;

CELL MEMBRANES;

GREEN FLUORESCENT PROTEIN; OCTAMER TRANSCRIPTION FACTOR 4; UNCLASSIFIED DRUG; ZINC FINGER NUCLEASE; ZINC FINGER PROTEIN;

ARTICLE; CONTROLLED STUDY; EMBRYO; EMBRYONIC STEM CELL; GENE EXPRESSION REGULATION; GENE INSERTION; GENE LOCUS; GENE OVEREXPRESSION; GENE TARGETING; HUMAN; HUMAN CELL; HUMAN GENOME; NUCLEOTIDE SEQUENCE; PLURIPOTENT STEM CELL; PRIORITY JOURNAL; PROMOTER REGION; SILENT GENE; TRANSGENE;

CELL LINE; DEOXYRIBONUCLEASES; EMBRYONIC STEM CELLS; GENE EXPRESSION; GENE SILENCING; GENE TARGETING; GREEN FLUORESCENT PROTEINS; HOMEODOMAIN PROTEINS; HUMANS; IMMUNOHISTOCHEMISTRY; OCTAMER TRANSCRIPTION FACTOR-3; PLURIPOTENT STEM CELLS; RECOMBINANT FUSION PROTEINS; TRANSCRIPTION FACTORS; ZINC FINGERS;

EID: 70249114821     PISSN: 10870156     EISSN: 15461696     Source Type: Journal    
DOI: 10.1038/nbt.1562     Document Type: Article

References (31)

1. Thomson, J.A. et al. embryonic stem cell lines derived from human blastocysts. Science 282, 1145-1147 (1998).
2. Urbach, A., Schuldiner, M. & Benvenisty, N. Modeling for lesch-Nyhan disease by gene targeting in human embryonic stem cells. Stem Cells 22, 635-641 (2004). (Pubitemid 39014579)
3. Costa, M. et al. A method for genetic modification of human embryonic stem cells using electroporation. Nat. Protoc. 2, 792-796 (2007).
4. Irion, S. et al. Identification and targeting of the ROSA26 locus in human embryonic stem cells. Nat. Biotechnol. 25, 1477-1482 (2007).
5. Suzuki, K. et al. Highly efficient transient gene expression and gene targeting in primate embryonic stem cells with helper-dependent adenoviral vectors. Proc. Natl. Acad. Sci. USA 105, 13781-13786 (2008).
6. Zwaka, T.P. & Thomson, J.A. Homologous recombination in human embryonic stem cells. Nat. Biotechnol. 21, 319-321 (2003).
7. Davis, R.P. et al. Targeting a GFP reporter gene to the MIXl1 locus of human embryonic stem cells identifies human primitive streak-like cells and enables isolation of primitive hematopoietic precursors. Blood 111, 1876-1884 (2008).
8. Xue, H. et al. A targeted neuroglial reporter line generated by homologous recombination in human embryonic stem cells. Stem Cells 27, 1836-1846 (2009).
9. Ruby, K.M. & Zheng, B. Gene targeting in a HUeS line of human embryonic stem cells via electroporation. Stem Cells 27, 1496-1506 (2009).
10. Urnov, F.D. et al. Highly efficient endogenous human gene correction using designed zinc-finger nucleases. Nature 435, 646-651 (2005).
11. Carroll, D. Progress and prospects: zinc-finger nucleases as gene therapy agents. Gene Ther. 15, 1463-1468 (2008).
12. Moehle, e.A. et al. Targeted gene addition into a specified location in the human genome using designed zinc finger nucleases. Proc. Natl. Acad. Sci. USA 104 3055-3060 2007).
13. Lombardo, A. et al. Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery. Nat. Biotechnol. 25, 1298-1306 (2007).
14. Cai, C.Q. et al. Targeted transgene integration in plant cells using designed zinc finger nucleases. Plant Mol. Biol. 69, 699-709 (2009).
15. Meng, X., Noyes, M.B., Zhu, l.J., lawson, N.D. & Wolfe, S.A. Targeted gene inac-tivation in zebrafish using engineered zinc-finger nucleases. Nat. Biotechnol. 26, 695-701 (2008).
16. Doyon, Y. et al. Heritable targeted gene disruption in zebrafish using designed zinc-finger nucleases. Nat. Biotechnol. 26, 702-708 (2008).
17. Shukla, V.K. et al. Precise genome modification in the crop species Zea mays using zinc-finger nucleases. Nature 459, 437-441 (2009).
18. Townsend, J.A. et al. High-frequency modification of plant genes using engineered zinc-finger nucleases. Nature 459, 442-445 (2009).
19. Geurts, A.M. et al. Knockout rats via embryo microinjection of zinc-finger nucleases. Science 325, 433 (2009).
20. Smith, J.R. et al. Robust, persistent transgene expression in human embryonic stem cells is achieved with AAVS1-targeted integration. Stem Cells 26, 496-504 (2008).
21. Soldner, F. et al. Parkinson?s disease patient-derived induced pluripotent stem cells free of viral reprogramming factors. Cell 136, 964-977 (2009).
22. Zou, J. et al. Gene targeting of a disease-related gene in human induced pluripotent stem and embryonic stem cells. Cell Stem Cell 5, 97-110 (2009).
23. Pabo, CO., Peisach, e. & Grant, R.A. Design and selection of novel Cys2His2 zinc finger proteins. Annu. Rev. Biochem. 70, 313-340 (2001).
24. Klug, A. The discovery of zinc fingers and their development for practical applications in gene regulation. Proc. Jpn. Acad. 81, 87-102 (2005).
25. + T cells by genome editing using zinc-finger nucleases. Nat. Biotechnol. 26 808-816 2008)
26. Miller, J.C. et al. An improved zinc-finger nuclease architecture for highly specific genome editing. Nat. Biotechnol. 25, 778-785 (2007).
27. Valerie, K. & Povirk, l.F. Regulation and mechanisms of mammalian doublestrand break repair. Oncogene 22, 5792-5812 (2003).
28. Bibikova, M., Golic, M., Golic, K.G. & Carroll, D. Targeted chromosomal cleavage and mutagenesis in Drosophila using zinc-finger nucleases. Genetics 161, 1169-1175 (2002).
29. Phillips, C.M. et al. Identification of chromosome sequence motifs that mediate meiotic pairing and synapsis in C. elegans. Nat. Cell. Biol. 11, 934-942 (2009).
30. Hockemeyer, D. et al. A drug-inducible system for direct reprogramming of human somatic cells to pluripotency. Cell Stem Cell 3, 346-353 (2008).
31. Brambrink, T. et al. Sequential expression of pluripotency markers during direct reprogramming of mouse somatic cells. Cell Stem Cell 2, 151-159 (2008).