메뉴 건너뛰기




Volumn 24, Issue 5, 2012, Pages 609-616

Development of nuclease-mediated site-specific genome modification

Author keywords

[No Author keywords available]

Indexed keywords

COMPLEMENTARY DNA; DNA; DNA FRAGMENT; NUCLEASE; NUCLEOTIDE; ZINC FINGER NUCLEASE;

EID: 84867743649     PISSN: 09527915     EISSN: 18790372     Source Type: Journal    
DOI: 10.1016/j.coi.2012.08.005     Document Type: Review
Times cited : (20)

References (75)
  • 3
    • 76249131912 scopus 로고    scopus 로고
    • Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease
    • Stein S., Ott M.G., Schultze-Strasser S., Jauch A., Burwinkel B., Kinner A., Schmidt M., Kramer A., Schwable J., Glimm H., et al. Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease. Nat Med 2010, 16:198-204.
    • (2010) Nat Med , vol.16 , pp. 198-204
    • Stein, S.1    Ott, M.G.2    Schultze-Strasser, S.3    Jauch, A.4    Burwinkel, B.5    Kinner, A.6    Schmidt, M.7    Kramer, A.8    Schwable, J.9    Glimm, H.10
  • 6
    • 70449704070 scopus 로고    scopus 로고
    • Progress and prospects: gene therapy for inherited immunodeficiencies
    • Qasim W., Gaspar H.B., Thrasher A.J. Progress and prospects: gene therapy for inherited immunodeficiencies. Gene Ther 2009, 16:1285-1291.
    • (2009) Gene Ther , vol.16 , pp. 1285-1291
    • Qasim, W.1    Gaspar, H.B.2    Thrasher, A.J.3
  • 8
    • 0038523969 scopus 로고    scopus 로고
    • Chimeric nucleases stimulate gene targeting in human cells
    • Porteus M.H., Baltimore D. Chimeric nucleases stimulate gene targeting in human cells. Science 2003, 300:763.
    • (2003) Science , vol.300 , pp. 763
    • Porteus, M.H.1    Baltimore, D.2
  • 9
    • 0024205183 scopus 로고
    • Targeted mutation of the Hprt gene in mouse embryonic stem cells
    • Doetschman T., Maeda N., Smithies O. Targeted mutation of the Hprt gene in mouse embryonic stem cells. Proc Natl Acad Sci U S A 1988, 85:8583-8587.
    • (1988) Proc Natl Acad Sci U S A , vol.85 , pp. 8583-8587
    • Doetschman, T.1    Maeda, N.2    Smithies, O.3
  • 10
    • 0023646810 scopus 로고
    • Site-directed mutagenesis by gene targeting in mouse embryo-derived stem cells
    • Thomas K.R., Capecchi M.R. Site-directed mutagenesis by gene targeting in mouse embryo-derived stem cells. Cell 1987, 51:503-512.
    • (1987) Cell , vol.51 , pp. 503-512
    • Thomas, K.R.1    Capecchi, M.R.2
  • 11
    • 0022389290 scopus 로고
    • Insertion of DNA sequences into the human chromosomal beta-globin locus by homologous recombination
    • Smithies O., Gregg R.G., Boggs S.S., Koralewski M.A., Kucherlapati R.S. Insertion of DNA sequences into the human chromosomal beta-globin locus by homologous recombination. Nature 1985, 317:230-234.
    • (1985) Nature , vol.317 , pp. 230-234
    • Smithies, O.1    Gregg, R.G.2    Boggs, S.S.3    Koralewski, M.A.4    Kucherlapati, R.S.5
  • 12
    • 0033032454 scopus 로고    scopus 로고
    • Gene targeting and somatic cell genetics-a rebirth or a coming of age?
    • Sedivy J.M., Dutriaux A. Gene targeting and somatic cell genetics-a rebirth or a coming of age?. Trends Genet 1999, 15:88-90.
    • (1999) Trends Genet , vol.15 , pp. 88-90
    • Sedivy, J.M.1    Dutriaux, A.2
  • 13
    • 0029867485 scopus 로고    scopus 로고
    • Stimulation of intrachromosomal homologous recombination in human cells by electroporation with site-specific endonucleases
    • Brenneman M., Gimble F.S., Wilson J.H. Stimulation of intrachromosomal homologous recombination in human cells by electroporation with site-specific endonucleases. Proc Natl Acad Sci U S A 1996, 93:3608-3612.
    • (1996) Proc Natl Acad Sci U S A , vol.93 , pp. 3608-3612
    • Brenneman, M.1    Gimble, F.S.2    Wilson, J.H.3
  • 14
    • 0028061666 scopus 로고
    • Introduction of double-strand breaks into the genome of mouse cells by expression of a rare-cutting endonuclease
    • Rouet P., Smih F., Jasin M. Introduction of double-strand breaks into the genome of mouse cells by expression of a rare-cutting endonuclease. Mol Cell Biol 1994, 14:8096-8106.
    • (1994) Mol Cell Biol , vol.14 , pp. 8096-8106
    • Rouet, P.1    Smih, F.2    Jasin, M.3
  • 15
    • 0028919608 scopus 로고
    • Induction of homologous recombination in mammalian chromosomes by using the I-SceI system of Saccharomyces cerevisiae
    • Choulika A., Perrin A., Dujon B., Nicolas J.F. Induction of homologous recombination in mammalian chromosomes by using the I-SceI system of Saccharomyces cerevisiae. Mol Cell Biol 1995, 15:1968-1973.
    • (1995) Mol Cell Biol , vol.15 , pp. 1968-1973
    • Choulika, A.1    Perrin, A.2    Dujon, B.3    Nicolas, J.F.4
  • 16
    • 0031863007 scopus 로고    scopus 로고
    • Analysis of gene targeting and intrachromosomal homologous recombination stimulated by genomic double-strand breaks in mouse embryonic stem cells
    • Donoho G., Jasin M., Berg P. Analysis of gene targeting and intrachromosomal homologous recombination stimulated by genomic double-strand breaks in mouse embryonic stem cells. Mol Cell Biol 1998, 18:4070-4078.
    • (1998) Mol Cell Biol , vol.18 , pp. 4070-4078
    • Donoho, G.1    Jasin, M.2    Berg, P.3
  • 17
    • 0024446859 scopus 로고
    • Group I introns as mobile genetic elements: facts and mechanistic speculations-a review
    • Dujon B. Group I introns as mobile genetic elements: facts and mechanistic speculations-a review. Gene 1989, 82:91-114.
    • (1989) Gene , vol.82 , pp. 91-114
    • Dujon, B.1
  • 18
    • 78651240053 scopus 로고    scopus 로고
    • Homing endonucleases: from microbial genetic invaders to reagents for targeted DNA modification
    • Stoddard B.L. Homing endonucleases: from microbial genetic invaders to reagents for targeted DNA modification. Structure 2011, 19:7-15.
    • (2011) Structure , vol.19 , pp. 7-15
    • Stoddard, B.L.1
  • 19
    • 33845604556 scopus 로고    scopus 로고
    • DNA double-strand break repair: all's well that ends well
    • Wyman C., Kanaar R. DNA double-strand break repair: all's well that ends well. Annu Rev Genet 2006, 40:363-383.
    • (2006) Annu Rev Genet , vol.40 , pp. 363-383
    • Wyman, C.1    Kanaar, R.2
  • 20
    • 3242886313 scopus 로고    scopus 로고
    • Homologous recombination-mediated double-strand break repair
    • Wyman C., Ristic D., Kanaar R. Homologous recombination-mediated double-strand break repair. DNA Repair (Amst) 2004, 3:827-833.
    • (2004) DNA Repair (Amst) , vol.3 , pp. 827-833
    • Wyman, C.1    Ristic, D.2    Kanaar, R.3
  • 21
    • 33847246298 scopus 로고    scopus 로고
    • Meganucleases and DNA double-strand break-induced recombination: perspectives for gene therapy
    • Paques F., Duchateau P. Meganucleases and DNA double-strand break-induced recombination: perspectives for gene therapy. Curr Gene Ther 2007, 7:49-66.
    • (2007) Curr Gene Ther , vol.7 , pp. 49-66
    • Paques, F.1    Duchateau, P.2
  • 22
    • 23444441147 scopus 로고    scopus 로고
    • Gene targeting using zinc finger nucleases
    • Porteus M.H., Carroll D. Gene targeting using zinc finger nucleases. Nat Biotechnol 2005, 23:967-973.
    • (2005) Nat Biotechnol , vol.23 , pp. 967-973
    • Porteus, M.H.1    Carroll, D.2
  • 23
    • 27744530147 scopus 로고    scopus 로고
    • Zinc finger nucleases: custom-designed molecular scissors for genome engineering of plant and mammalian cells
    • Durai S., Mani M., Kandavelou K., Wu J., Porteus M.H., Chandrasegaran S. Zinc finger nucleases: custom-designed molecular scissors for genome engineering of plant and mammalian cells. Nucleic Acids Res 2005, 33:5978-5990.
    • (2005) Nucleic Acids Res , vol.33 , pp. 5978-5990
    • Durai, S.1    Mani, M.2    Kandavelou, K.3    Wu, J.4    Porteus, M.H.5    Chandrasegaran, S.6
  • 24
    • 80053343092 scopus 로고    scopus 로고
    • TAL effectors: customizable proteins for DNA targeting
    • Bogdanove A.J., Voytas D.F. TAL effectors: customizable proteins for DNA targeting. Science 2011, 333:1843-1846.
    • (2011) Science , vol.333 , pp. 1843-1846
    • Bogdanove, A.J.1    Voytas, D.F.2
  • 29
    • 41149127259 scopus 로고    scopus 로고
    • Comparison of zinc finger nucleases for use in gene targeting in mammalian cells
    • Pruett-Miller S.M., Connelly J.P., Maeder M.L., Joung J.K., Porteus M.H. Comparison of zinc finger nucleases for use in gene targeting in mammalian cells. Mol Ther 2008, 16:707-717.
    • (2008) Mol Ther , vol.16 , pp. 707-717
    • Pruett-Miller, S.M.1    Connelly, J.P.2    Maeder, M.L.3    Joung, J.K.4    Porteus, M.H.5
  • 31
    • 45749145124 scopus 로고    scopus 로고
    • Zinc-finger nucleases: the next generation emerges
    • Cathomen T., Joung J.K. Zinc-finger nucleases: the next generation emerges. Mol Ther 2008, 16:1200-1207.
    • (2008) Mol Ther , vol.16 , pp. 1200-1207
    • Cathomen, T.1    Joung, J.K.2
  • 32
    • 0030032063 scopus 로고    scopus 로고
    • Hybrid restriction enzymes: zinc finger fusions to Fok I cleavage domain
    • Kim Y.G., Cha J., Chandrasegaran S. Hybrid restriction enzymes: zinc finger fusions to Fok I cleavage domain. Proc Natl Acad Sci U S A 1996, 93:1156-1160.
    • (1996) Proc Natl Acad Sci U S A , vol.93 , pp. 1156-1160
    • Kim, Y.G.1    Cha, J.2    Chandrasegaran, S.3
  • 33
    • 0034283804 scopus 로고    scopus 로고
    • Requirements for double-strand cleavage by chimeric restriction enzymes with zinc finger DNA-recognition domains
    • Smith J., Bibikova M., Whitby F.G., Reddy A.R., Chandrasegaran S., Carroll D. Requirements for double-strand cleavage by chimeric restriction enzymes with zinc finger DNA-recognition domains. Nucleic Acids Res 2000, 28:3361-3369.
    • (2000) Nucleic Acids Res , vol.28 , pp. 3361-3369
    • Smith, J.1    Bibikova, M.2    Whitby, F.G.3    Reddy, A.R.4    Chandrasegaran, S.5    Carroll, D.6
  • 34
    • 30344473645 scopus 로고    scopus 로고
    • Mammalian gene targeting with designed zinc finger nucleases
    • Porteus M.H. Mammalian gene targeting with designed zinc finger nucleases. Mol Ther 2006, 13:438-446.
    • (2006) Mol Ther , vol.13 , pp. 438-446
    • Porteus, M.H.1
  • 37
    • 0035126270 scopus 로고    scopus 로고
    • Severe combined immunodeficiency-molecular pathogenesis and diagnosis
    • Gaspar H.B., Gilmour K.C., Jones A.M. Severe combined immunodeficiency-molecular pathogenesis and diagnosis. Arch Dis Child 2001, 84:169-173.
    • (2001) Arch Dis Child , vol.84 , pp. 169-173
    • Gaspar, H.B.1    Gilmour, K.C.2    Jones, A.M.3
  • 38
    • 79957587075 scopus 로고    scopus 로고
    • Oxidase-deficient neutrophils from X-linked chronic granulomatous disease iPS cells: functional correction by zinc finger nuclease-mediated safe harbor targeting
    • Zou J., Sweeney C.L., Chou B.K., Choi U., Pan J., Wang H., Dowey S.N., Cheng L., Malech H.L. Oxidase-deficient neutrophils from X-linked chronic granulomatous disease iPS cells: functional correction by zinc finger nuclease-mediated safe harbor targeting. Blood 2011, 117:5561-5572.
    • (2011) Blood , vol.117 , pp. 5561-5572
    • Zou, J.1    Sweeney, C.L.2    Chou, B.K.3    Choi, U.4    Pan, J.5    Wang, H.6    Dowey, S.N.7    Cheng, L.8    Malech, H.L.9
  • 39
    • 77955157281 scopus 로고    scopus 로고
    • Functional genomics, proteomics, and regulatory DNA analysis in isogenic settings using zinc finger nuclease-driven transgenesis into a safe harbor locus in the human genome
    • DeKelver R.C., Choi V.M., Moehle E.A., Paschon D.E., Hockemeyer D., Meijsing S.H., Sancak Y., Cui X., Steine E.J., Miller J.C., et al. Functional genomics, proteomics, and regulatory DNA analysis in isogenic settings using zinc finger nuclease-driven transgenesis into a safe harbor locus in the human genome. Genome Res 2010, 20:1133-1142.
    • (2010) Genome Res , vol.20 , pp. 1133-1142
    • DeKelver, R.C.1    Choi, V.M.2    Moehle, E.A.3    Paschon, D.E.4    Hockemeyer, D.5    Meijsing, S.H.6    Sancak, Y.7    Cui, X.8    Steine, E.J.9    Miller, J.C.10
  • 41
    • 50149108172 scopus 로고    scopus 로고
    • The promise of human induced pluripotent stem cells for research and therapy
    • Nishikawa S., Goldstein R.A., Nierras C.R. The promise of human induced pluripotent stem cells for research and therapy. Nat Rev Mol Cell Biol 2008, 9:725-729.
    • (2008) Nat Rev Mol Cell Biol , vol.9 , pp. 725-729
    • Nishikawa, S.1    Goldstein, R.A.2    Nierras, C.R.3
  • 42
    • 80255126235 scopus 로고    scopus 로고
    • Primary immunodeficiency modeling with induced pluripotent stem cells
    • Pessach I.M., Notarangelo L.D. Primary immunodeficiency modeling with induced pluripotent stem cells. Curr Opin Allergy Clin Immunol 2011, 11:505-511.
    • (2011) Curr Opin Allergy Clin Immunol , vol.11 , pp. 505-511
    • Pessach, I.M.1    Notarangelo, L.D.2
  • 44
    • 80055069793 scopus 로고    scopus 로고
    • Site-specific gene correction of a point mutation in human iPS cells derived from an adult patient with sickle cell disease
    • Zou J., Mali P., Huang X., Dowey S.N., Cheng L. Site-specific gene correction of a point mutation in human iPS cells derived from an adult patient with sickle cell disease. Blood 2011, 118:4599-4608.
    • (2011) Blood , vol.118 , pp. 4599-4608
    • Zou, J.1    Mali, P.2    Huang, X.3    Dowey, S.N.4    Cheng, L.5
  • 48
    • 78650971216 scopus 로고    scopus 로고
    • Dynamic changes in the copy number of pluripotency and cell proliferation genes in human ESCs and iPSCs during reprogramming and time in culture
    • Laurent L.C., Ulitsky I., Slavin I., Tran H., Schork A., Morey R., Lynch C., Harness J.V., Lee S., Barrero M.J., et al. Dynamic changes in the copy number of pluripotency and cell proliferation genes in human ESCs and iPSCs during reprogramming and time in culture. Cell Stem Cell 2011, 8:106-118.
    • (2011) Cell Stem Cell , vol.8 , pp. 106-118
    • Laurent, L.C.1    Ulitsky, I.2    Slavin, I.3    Tran, H.4    Schork, A.5    Morey, R.6    Lynch, C.7    Harness, J.V.8    Lee, S.9    Barrero, M.J.10
  • 50
    • 79957807595 scopus 로고    scopus 로고
    • Immunogenicity of induced pluripotent stem cells
    • Zhao T., Zhang Z.N., Rong Z., Xu Y. Immunogenicity of induced pluripotent stem cells. Nature 2011, 474:212-215.
    • (2011) Nature , vol.474 , pp. 212-215
    • Zhao, T.1    Zhang, Z.N.2    Rong, Z.3    Xu, Y.4
  • 52
    • 77956294479 scopus 로고    scopus 로고
    • Generation of genetically modified rats from embryonic stem cells
    • Kawamata M., Ochiya T. Generation of genetically modified rats from embryonic stem cells. Proc Natl Acad Sci U S A 2010, 107:14223-14228.
    • (2010) Proc Natl Acad Sci U S A , vol.107 , pp. 14223-14228
    • Kawamata, M.1    Ochiya, T.2
  • 53
    • 77956524118 scopus 로고    scopus 로고
    • Production of p53 gene knockout rats by homologous recombination in embryonic stem cells
    • Tong C., Li P., Wu N.L., Yan Y., Ying Q.L. Production of p53 gene knockout rats by homologous recombination in embryonic stem cells. Nature 2010, 467:211-213.
    • (2010) Nature , vol.467 , pp. 211-213
    • Tong, C.1    Li, P.2    Wu, N.L.3    Yan, Y.4    Ying, Q.L.5
  • 54
    • 77957005768 scopus 로고    scopus 로고
    • Gene targeting by homologous recombination in mouse zygotes mediated by zinc-finger nucleases
    • Meyer M., de Angelis M.H., Wurst W., Kuhn R. Gene targeting by homologous recombination in mouse zygotes mediated by zinc-finger nucleases. Proc Natl Acad Sci U S A 2010, 107:15022-15026.
    • (2010) Proc Natl Acad Sci U S A , vol.107 , pp. 15022-15026
    • Meyer, M.1    de Angelis, M.H.2    Wurst, W.3    Kuhn, R.4
  • 58
  • 59
    • 77749273530 scopus 로고    scopus 로고
    • Generation of knockout rats with X-linked severe combined immunodeficiency (X-SCID) using zinc-finger nucleases
    • Mashimo T., Takizawa A., Voigt B., Yoshimi K., Hiai H., Kuramoto T., Serikawa T. Generation of knockout rats with X-linked severe combined immunodeficiency (X-SCID) using zinc-finger nucleases. PLoS One 2010, 5:e8870.
    • (2010) PLoS One , vol.5
    • Mashimo, T.1    Takizawa, A.2    Voigt, B.3    Yoshimi, K.4    Hiai, H.5    Kuramoto, T.6    Serikawa, T.7
  • 63
    • 80053039555 scopus 로고    scopus 로고
    • A novel TALE nuclease scaffold enables high genome editing activity in combination with low toxicity
    • Mussolino C., Morbitzer R., Lutge F., Dannemann N., Lahaye T., Cathomen T. A novel TALE nuclease scaffold enables high genome editing activity in combination with low toxicity. Nucleic Acids Res 2011, 39:9283-9293.
    • (2011) Nucleic Acids Res , vol.39 , pp. 9283-9293
    • Mussolino, C.1    Morbitzer, R.2    Lutge, F.3    Dannemann, N.4    Lahaye, T.5    Cathomen, T.6
  • 65
    • 80052293623 scopus 로고    scopus 로고
    • Revealing off-target cleavage specificities of zinc-finger nucleases by in vitro selection
    • Pattanayak V., Ramirez C.L., Joung J.K., Liu D.R. Revealing off-target cleavage specificities of zinc-finger nucleases by in vitro selection. Nat Methods 2011, 8:765-770.
    • (2011) Nat Methods , vol.8 , pp. 765-770
    • Pattanayak, V.1    Ramirez, C.L.2    Joung, J.K.3    Liu, D.R.4
  • 66
    • 34447323568 scopus 로고    scopus 로고
    • Structure-based redesign of the dimerization interface reduces the toxicity of zinc-finger nucleases
    • Szczepek M., Brondani V., Buchel J., Serrano L., Segal D.J., Cathomen T. Structure-based redesign of the dimerization interface reduces the toxicity of zinc-finger nucleases. Nat Biotechnol 2007, 25:786-793.
    • (2007) Nat Biotechnol , vol.25 , pp. 786-793
    • Szczepek, M.1    Brondani, V.2    Buchel, J.3    Serrano, L.4    Segal, D.J.5    Cathomen, T.6
  • 69
    • 61449266636 scopus 로고    scopus 로고
    • Attenuation of zinc finger nuclease toxicity by small-molecule regulation of protein levels
    • Pruett-Miller S.M., Reading D.W., Porter S.N., Porteus M.H. Attenuation of zinc finger nuclease toxicity by small-molecule regulation of protein levels. PLoS Genet 2009, 5:e1000376.
    • (2009) PLoS Genet , vol.5
    • Pruett-Miller, S.M.1    Reading, D.W.2    Porter, S.N.3    Porteus, M.H.4
  • 70
    • 79960034141 scopus 로고    scopus 로고
    • Modularly assembled designer TAL effector nucleases for targeted gene knockout and gene replacement in eukaryotes
    • Li T., Huang S., Zhao X., Wright D.A., Carpenter S., Spalding M.H., Weeks D.P., Yang B. Modularly assembled designer TAL effector nucleases for targeted gene knockout and gene replacement in eukaryotes. Nucleic Acids Res 2011, 39:6315-6325.
    • (2011) Nucleic Acids Res , vol.39 , pp. 6315-6325
    • Li, T.1    Huang, S.2    Zhao, X.3    Wright, D.A.4    Carpenter, S.5    Spalding, M.H.6    Weeks, D.P.7    Yang, B.8
  • 71
    • 79961185942 scopus 로고    scopus 로고
    • Heritable gene targeting in zebrafish using customized TALENs
    • Huang P., Xiao A., Zhou M., Zhu Z., Lin S., Zhang B. Heritable gene targeting in zebrafish using customized TALENs. Nat Biotechnol 2011, 29:699-700.
    • (2011) Nat Biotechnol , vol.29 , pp. 699-700
    • Huang, P.1    Xiao, A.2    Zhou, M.3    Zhu, Z.4    Lin, S.5    Zhang, B.6
  • 72
  • 73
    • 84857032466 scopus 로고    scopus 로고
    • Structural basis for sequence-specific recognition of DNA by TAL effectors
    • Deng D., Yan C., Pan X., Mahfouz M., Wang J., Zhu J.K., Shi Y., Yan N. Structural basis for sequence-specific recognition of DNA by TAL effectors. Science 2012, 335:720-723.
    • (2012) Science , vol.335 , pp. 720-723
    • Deng, D.1    Yan, C.2    Pan, X.3    Mahfouz, M.4    Wang, J.5    Zhu, J.K.6    Shi, Y.7    Yan, N.8
  • 74


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.