메뉴 건너뛰기




Volumn 435, Issue 7042, 2005, Pages 646-651

Highly efficient endogenous human gene correction using designed zinc-finger nucleases

Author keywords

[No Author keywords available]

Indexed keywords

BIOMEDICAL ENGINEERING; CELLS; DISEASE CONTROL; DISEASES; DNA; GENES; IMMUNOLOGY; PROTEINS; RNA;

EID: 18944373328     PISSN: 00280836     EISSN: None     Source Type: Journal    
DOI: 10.1038/nature03556     Document Type: Article
Times cited : (1414)

References (39)
  • 2
    • 0031935394 scopus 로고    scopus 로고
    • Therapeutic gene targeting
    • Yanez, R. J. & Porter, A. C. Therapeutic gene targeting. Gene Ther. 5, 149-159 (1998).
    • (1998) Gene Ther. , vol.5 , pp. 149-159
    • Yanez, R.J.1    Porter, A.C.2
  • 3
    • 0042738935 scopus 로고    scopus 로고
    • Occurrence of leukaemia following gene therapy of X-linked SCID
    • Kohn, D. B., Sadelain, M. & Glorioso, J. C. Occurrence of leukaemia following gene therapy of X-linked SCID. Nature Rev. Cancer 3, 477-488 (2003).
    • (2003) Nature Rev. Cancer , vol.3 , pp. 477-488
    • Kohn, D.B.1    Sadelain, M.2    Glorioso, J.C.3
  • 4
    • 0141990752 scopus 로고    scopus 로고
    • Gene therapy for the hemoglobin disorders
    • Persons, D. A. & Nienhuis, A. W. Gene therapy for the hemoglobin disorders, Curr. Hematol. Rep. 2, 348-355 (2003).
    • (2003) Curr. Hematol. Rep. , vol.2 , pp. 348-355
    • Persons, D.A.1    Nienhuis, A.W.2
  • 5
    • 0022542596 scopus 로고
    • High frequency targeting of genes to specific sites in the mammalian genome
    • Thomas, K. R., Folger, K. R. & Capecchi, M. R. High frequency targeting of genes to specific sites in the mammalian genome. Cell 44, 419-428 (1986).
    • (1986) Cell , vol.44 , pp. 419-428
    • Thomas, K.R.1    Folger, K.R.2    Capecchi, M.R.3
  • 6
    • 0030609861 scopus 로고    scopus 로고
    • Bypass of senescence after disruption of P21CIP1/WAF1 gene in normal diploid human fibroblasts
    • Brown, J. P., Wei, W. & Sedivy, J. M. Bypass of senescence after disruption of P21CIP1/WAF1 gene in normal diploid human fibroblasts. Science 277, 831-834 (1997).
    • (1997) Science , vol.277 , pp. 831-834
    • Brown, J.P.1    Wei, W.2    Sedivy, J.M.3
  • 7
    • 0036169078 scopus 로고    scopus 로고
    • Targeted inactivation of p53 in human cells does not result in aneuploidy
    • Bunz, F. et al. Targeted inactivation of p53 in human cells does not result in aneuploidy. Cancer Res. 62, 1129-1133 (2002).
    • (2002) Cancer Res. , vol.62 , pp. 1129-1133
    • Bunz, F.1
  • 8
    • 0040215628 scopus 로고
    • Repetitive zinc-binding domains in the protein transcription factor IIIA from Xenopus oocytes
    • Miller, J., McLachlan, A. D. & Klug, A. Repetitive zinc-binding domains in the protein transcription factor IIIA from Xenopus oocytes. EMBO J. 4, 1609-1614 (1985).
    • (1985) EMBO J. , vol.4 , pp. 1609-1614
    • Miller, J.1    McLachlan, A.D.2    Klug, A.3
  • 9
    • 0035865250 scopus 로고    scopus 로고
    • Expressing the human genome
    • Tupler, R., Perini, G. & Green, M. R. Expressing the human genome. Nature 409, 832-833 (2001).
    • (2001) Nature , vol.409 , pp. 832-833
    • Tupler, R.1    Perini, G.2    Green, M.R.3
  • 10
    • 0025773296 scopus 로고
    • Zinc finger-DNA recognition: Crystal structure of a Zif268-DNA complex at 2.1 Å
    • Pavletich, N. P. & Pabo, C. O. Zinc finger-DNA recognition: crystal structure of a Zif268-DNA complex at 2.1 Å. Science 252, 809-817 (1991).
    • (1991) Science , vol.252 , pp. 809-817
    • Pavletich, N.P.1    Pabo, C.O.2
  • 11
    • 0027767784 scopus 로고
    • Protein designs for the specific recognition of DNA
    • Klug, A. Protein designs for the specific recognition of DNA. Gene 135, 83-92 (1993).
    • (1993) Gene , vol.135 , pp. 83-92
    • Klug, A.1
  • 12
    • 0028670569 scopus 로고
    • In vivo repression by a site-specific DNA-binding protein designed against an oncogenic sequence
    • Choo, Y., Sanchez-Garcia, I. & Klug, A. In vivo repression by a site-specific DNA-binding protein designed against an oncogenic sequence. Nature 372, 642-645 (1994).
    • (1994) Nature , vol.372 , pp. 642-645
    • Choo, Y.1    Sanchez-Garcia, I.2    Klug, A.3
  • 13
    • 0034923498 scopus 로고    scopus 로고
    • Design and selection of novel Cys 2his2 zinc finger proteins
    • Pabo, C. O., Peisach, E. & Grant, R. A. Design and selection of novel Cys2his2 zinc finger proteins. Annu. Rev. Biochem. 70, 313-340 (2001).
    • (2001) Annu. Rev. Biochem. , vol.70 , pp. 313-340
    • Pabo, C.O.1    Peisach, E.2    Grant, R.A.3
  • 14
    • 0033930952 scopus 로고    scopus 로고
    • Advances in zinc finger engineering
    • Choo, Y. & Isalan, M. Advances in zinc finger engineering. Curr. Opin. Struct. Biol. 10, 411-416 (2000).
    • (2000) Curr. Opin. Struct. Biol. , vol.10 , pp. 411-416
    • Choo, Y.1    Isalan, M.2
  • 16
    • 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. USA 93, 1156-1160 (1996).
    • (1996) Proc. Natl. Acad. Sci. USA , vol.93 , pp. 1156-1160
    • Kim, Y.G.1    Cha, J.2    Chandrasegaran, S.3
  • 17
    • 0033556128 scopus 로고    scopus 로고
    • A detailed study of the substrate specificity of a chimeric restriction enzyme
    • Smith, J., Berg, J. M. & Chandrasegaran, S. A detailed study of the substrate specificity of a chimeric restriction enzyme. Nucleic Acids Res. 27, 674-681 (1999).
    • (1999) Nucleic Acids Res. , vol.27 , pp. 674-681
    • Smith, J.1    Berg, J.M.2    Chandrasegaran, S.3
  • 18
    • 0038700698 scopus 로고    scopus 로고
    • Molecular views of recombination proteins and their control
    • West, S. C. Molecular views of recombination proteins and their control. Nature Rev. Mol. Cell Biol. 4, 435-445 (2003).
    • (2003) Nature Rev. Mol. Cell Biol. , vol.4 , pp. 435-445
    • West, S.C.1
  • 19
    • 0036900120 scopus 로고    scopus 로고
    • Role of RAD52 epistasis group genes in homologous recombination and double-strand break repair
    • Symington, L. S. Role of RAD52 epistasis group genes in homologous recombination and double-strand break repair. Microbiol. Mol. Biol. Rev. 66, 630-670 (2002).
    • (2002) Microbiol. Mol. Biol. Rev. , vol.66 , pp. 630-670
    • Symington, L.S.1
  • 20
    • 0038799991 scopus 로고    scopus 로고
    • Multiple pathways of recombination induced by double-strand breaks in Saccharomyces cerevisiae
    • Paques, F. & Haber, J. E. Multiple pathways of recombination induced by double-strand breaks in Saccharomyces cerevisiae. Microbiol. Mol. Biol. Rev. 63, 349-404 (1999).
    • (1999) Microbiol. Mol. Biol. Rev. , vol.63 , pp. 349-404
    • Paques, F.1    Haber, J.E.2
  • 21
    • 0028237305 scopus 로고
    • Expression of a site-specific endonuclease stimulates homologous recombination in mammalian cells
    • Rouet, P., Smih, F. & Jasin, M. Expression of a site-specific endonuclease stimulates homologous recombination in mammalian cells. Proc. Natl Acad. Sci. USA 91, 6064-6068 (1994).
    • (1994) Proc. Natl. Acad. Sci. USA , vol.91 , pp. 6064-6068
    • Rouet, P.1    Smih, F.2    Jasin, M.3
  • 22
    • 0031032549 scopus 로고    scopus 로고
    • Repair of site-specific double-strand breaks in a mammalian chromosome by homologous and illegitimate recombination
    • Sargent, R. G., Brenneman, M. A. & Wilson, J. H. Repair of site-specific double-strand breaks in a mammalian chromosome by homologous and illegitimate recombination. Mol. Cell. Biol. 17, 267-277 (1997).
    • (1997) Mol. Cell. Biol. , vol.17 , pp. 267-277
    • Sargent, R.G.1    Brenneman, M.A.2    Wilson, J.H.3
  • 23
    • 0028919608 scopus 로고
    • Induction of homologous recombination in mammalian chromosomes by using the I-Scel system of Saccharomyces cerevisiae
    • Choulika, A., Perrin, A., Dujon, B. & Nicolas, J. F. Induction of homologous recombination in mammalian chromosomes by using the I-Scel system of Saccharomyces cerevisiae. Mol. Cell. Biol. 15, 1968-1973 (1995).
    • (1995) Mol. Cell. Biol. , vol.15 , pp. 1968-1973
    • Choulika, A.1    Perrin, A.2    Dujon, B.3    Nicolas, J.F.4
  • 24
    • 0034749283 scopus 로고    scopus 로고
    • Stimulation of homologous recombination through targeted cleavage by chimeric nucleases
    • Bibikova, M. et al. Stimulation of homologous recombination through targeted cleavage by chimeric nucleases. Mol. Cell. Biol. 21, 289-297 (2001).
    • (2001) Mol. Cell. Biol. , vol.21 , pp. 289-297
    • Bibikova, M.1
  • 25
    • 0037510038 scopus 로고    scopus 로고
    • Enhancing gene targeting with designed zinc finger nucleases
    • Bibikova, M., Beumer, K., Trautman, J. K. & Carroll, D. Enhancing gene targeting with designed zinc finger nucleases. Science 300, 764 (2003).
    • (2003) Science , vol.300 , pp. 764
    • Bibikova, M.1    Beumer, K.2    Trautman, J.K.3    Carroll, D.4
  • 26
    • 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 300, 763 (2003).
    • (2003) Science , vol.300 , pp. 763
    • Porteus, M.H.1    Baltimore, D.2
  • 27
    • 0034724857 scopus 로고    scopus 로고
    • Gene therapy of human severe combined immunodeficiency (SCID)-X 1 disease
    • Cavazzana-Calvo, M. et al. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 288, 669-672 (2000).
    • (2000) Science , vol.288 , pp. 669-672
    • Cavazzana-Calvo, M.1
  • 29
    • 0035852739 scopus 로고    scopus 로고
    • Improved DNA binding specificity from polyzinc finger peptides by using strings of two-finger units
    • Moore, M., Klug, A. & Choo, Y. Improved DNA binding specificity from polyzinc finger peptides by using strings of two-finger units. Proc. Natl Acad. Sci. USA 98, 1437-1441 (2001).
    • (2001) Proc. Natl. Acad. Sci. USA , vol.98 , pp. 1437-1441
    • Moore, M.1    Klug, A.2    Choo, Y.3
  • 30
    • 0034597508 scopus 로고    scopus 로고
    • Primary immunodeficiency diseases due to defects in lymphocytes
    • Buckley, R. H. Primary immunodeficiency diseases due to defects in lymphocytes. N. Engl. J. Med. 343, 1313-1324 (2000).
    • (2000) N. Engl. J. Med. , vol.343 , pp. 1313-1324
    • Buckley, R.H.1
  • 31
    • 0036204810 scopus 로고    scopus 로고
    • Integration of splicing, transport and translation to achieve mRNA quality control by the nonsense-mediated decay pathway
    • Schell, T., Kulozik, A. E. & Hentze, M. W. Integration of splicing, transport and translation to achieve mRNA quality control by the nonsense-mediated decay pathway. Genome Biol. 3, 1-6 (2002).
    • (2002) Genome Biol. , vol.3 , pp. 1-6
    • Schell, T.1    Kulozik, A.E.2    Hentze, M.W.3
  • 32
    • 0034887244 scopus 로고    scopus 로고
    • Rapid, high-throughput engineering of sequence-specific zinc finger DNA-binding proteins
    • Isalan, M. & Choo, Y. Rapid, high-throughput engineering of sequence-specific zinc finger DNA-binding proteins. Methods Enzymol. 340, 593-609 (2001).
    • (2001) Methods Enzymol. , vol.340 , pp. 593-609
    • Isalan, M.1    Choo, Y.2
  • 33
    • 0037716265 scopus 로고    scopus 로고
    • Pointing fingers at the limiting step in gene targeting
    • Wilson, J. H. Pointing fingers at the limiting step in gene targeting. Nature Biotechnol. 21, 759-760 (2003).
    • (2003) Nature Biotechnol. , vol.21 , pp. 759-760
    • Wilson, J.H.1
  • 34
    • 0031042082 scopus 로고    scopus 로고
    • Physical basis of a protein-DNA recognition code
    • Choo, Y. & Klug, A. Physical basis of a protein-DNA recognition code. Curr. Opin. Struct. Biol. 7, 117-125 (1997).
    • (1997) Curr. Opin. Struct. Biol. , vol.7 , pp. 117-125
    • Choo, Y.1    Klug, A.2
  • 35
    • 0036021389 scopus 로고    scopus 로고
    • Targeted chromosomal cleavage and mutagenesis in Drosophila using zinc-finger nucleases
    • 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).
    • (2002) Genetics , vol.161 , pp. 1169-1175
    • Bibikova, M.1    Golic, M.2    Golic, K.G.3    Carroll, D.4
  • 36
    • 0034721877 scopus 로고    scopus 로고
    • Synthetic zinc finger transcription factor action at an endogenous chromosomal site. Activation of the human erythropoietin gene
    • Zhang, L. et al. Synthetic zinc finger transcription factor action at an endogenous chromosomal site. Activation of the human erythropoietin gene. J. Biol. Chem. 275, 33850-33860 (2000).
    • (2000) J. Biol. Chem. , vol.275 , pp. 33850-33860
    • Zhang, L.1
  • 37
    • 0035815697 scopus 로고    scopus 로고
    • Regulation of an endogenous locus using a panel of designed zinc finger proteins targeted to accessible chromatin regions. Activation of vascular endothelial growth factor A
    • Liu, P. Q. et al. Regulation of an endogenous locus using a panel of designed zinc finger proteins targeted to accessible chromatin regions. Activation of vascular endothelial growth factor A. J. Biol. Chem. 276, 11323-11334 (2001).
    • (2001) J. Biol. Chem. , vol.276 , pp. 11323-11334
    • Liu, P.Q.1
  • 38
    • 0034930363 scopus 로고    scopus 로고
    • A rapid, generally applicable method to engineer zinc fingers illustrated by targeting the HIV-1 promoter
    • Isalan, M., Klug, A. & Choo, Y. A rapid, generally applicable method to engineer zinc fingers illustrated by targeting the HIV-1 promoter. Nature Biotechnol. 19, 656-660 (2001).
    • (2001) Nature Biotechnol. , vol.19 , pp. 656-660
    • Isalan, M.1    Klug, A.2    Choo, Y.3
  • 39
    • 0142027810 scopus 로고    scopus 로고
    • Zinc-finger protein-targeted gene regulation: Genomewide single-gene specificity
    • Tan, S. et al. Zinc-finger protein-targeted gene regulation: genomewide single-gene specificity. Proc. Natl Acad. Sci. USA 100, 11997-12002 (2003).
    • (2003) Proc. Natl. Acad. Sci. USA , vol.100 , pp. 11997-12002
    • Tan, S.1


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.