메뉴 건너뛰기




Volumn 6, Issue 4, 2013, Pages 896-904

Genome editing of human pluripotent stem cells to generate human cellular disease models

Author keywords

[No Author keywords available]

Indexed keywords

ESTERASE; MEGANUCLEASE; TRANSCRIPTION ACTIVATOR LIKE EFFECTOR NUCLEASE; UNCLASSIFIED DRUG; ZINC FINGER NUCLEASE;

EID: 84880026566     PISSN: 17548403     EISSN: 17548411     Source Type: Journal    
DOI: 10.1242/dmm.012054     Document Type: Review
Times cited : (127)

References (93)
  • 1
    • 83255189758 scopus 로고    scopus 로고
    • Screening ethnically diverse human embryonic stem cells identifies a chromosome 20 minimal amplicon conferring growth advantage
    • International Stem Cell Initiative
    • Amps, K., Andrews, P. W., Anyfantis, G., Armstrong, L., Avery, S., Baharvand, H., Baker, J., Baker, D., Munoz, M. B., Beil, S. et al.; International Stem Cell Initiative (2011). Screening ethnically diverse human embryonic stem cells identifies a chromosome 20 minimal amplicon conferring growth advantage. Nat. Biotechnol. 29, 1132-1144.
    • (2011) Nat. Biotechnol. , vol.29 , pp. 1132-1144
    • Amps, K.1    Andrews, P.W.2    Anyfantis, G.3    Armstrong, L.4    Avery, S.5    Baharvand, H.6    Baker, J.7    Baker, D.8    Munoz, M.B.9    Beil, S.10
  • 3
    • 79959992876 scopus 로고    scopus 로고
    • Epigenetic memory and preferential lineage-specific differentiation in induced pluripotent stem cells derived from human pancreatic islet beta cells
    • Bar-Nur, O., Russ, H. A., Efrat, S. and Benvenisty, N. (2011). Epigenetic memory and preferential lineage-specific differentiation in induced pluripotent stem cells derived from human pancreatic islet beta cells. Cell Stem Cell 9, 17-23.
    • (2011) Cell Stem Cell , vol.9 , pp. 17-23
    • Bar-Nur, O.1    Russ, H.A.2    Efrat, S.3    Benvenisty, N.4
  • 6
  • 7
    • 80053343092 scopus 로고    scopus 로고
    • TAL effectors: Customizable proteins for DNA targeting
    • Bogdanove, A. J. and Voytas, D. F. (2011). TAL effectors: customizable proteins for DNA targeting. Science 333, 1843-1846.
    • (2011) Science , vol.333 , pp. 1843-1846
    • Bogdanove, A.J.1    Voytas, D.F.2
  • 9
    • 84863201964 scopus 로고    scopus 로고
    • Targeted transcriptional activation of silent oct4 pluripotency gene by combining designer TALEs and inhibition of epigenetic modifiers
    • Bultmann, S., Morbitzer, R., Schmidt, C. S., Thanisch, K., Spada, F., Elsaesser, J., Lahaye, T. and Leonhardt, H. (2012). Targeted transcriptional activation of silent oct4 pluripotency gene by combining designer TALEs and inhibition of epigenetic modifiers. Nucleic Acids Res. 40, 5368-5377.
    • (2012) Nucleic Acids Res. , vol.40 , pp. 5368-5377
    • Bultmann, S.1    Morbitzer, R.2    Schmidt, C.S.3    Thanisch, K.4    Spada, F.5    Elsaesser, J.6    Lahaye, T.7    Leonhardt, H.8
  • 12
    • 84874624936 scopus 로고    scopus 로고
    • Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease
    • Cho, S. W., Kim, S., Kim, J. M. and Kim, J. S. (2013). Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease. Nat. Biotechnol. 31, 230-232.
    • (2013) Nat. Biotechnol. , vol.31 , pp. 230-232
    • Cho, S.W.1    Kim, S.2    Kim, J.M.3    Kim, J.S.4
  • 16
    • 56549096129 scopus 로고    scopus 로고
    • Human embryonic stem cell-derived motor neurons are sensitive to the toxic effect of glial cells carrying an ALS-causing mutation
    • Di Giorgio, F. P., Boulting, G. L., Bobrowicz, S. and Eggan, K. C. (2008). Human embryonic stem cell-derived motor neurons are sensitive to the toxic effect of glial cells carrying an ALS-causing mutation. Cell Stem Cell 3, 637-648.
    • (2008) Cell Stem Cell , vol.3 , pp. 637-648
    • Di Giorgio, F.P.1    Boulting, G.L.2    Bobrowicz, S.3    Eggan, K.C.4
  • 24
    • 84864628471 scopus 로고    scopus 로고
    • Induced pluripotent stem cells from patients with Huntington's disease show CAG-repeat-expansion-associated phenotypes
    • HD iPSC Consortium
    • HD iPSC Consortium (2012). Induced pluripotent stem cells from patients with Huntington's disease show CAG-repeat-expansion-associated phenotypes. Cell Stem Cell 11, 264-278.
    • (2012) Cell Stem Cell , vol.11 , pp. 264-278
  • 29
    • 84857496469 scopus 로고    scopus 로고
    • Elevated coding mutation rate during the reprogramming of human somatic cells into induced pluripotent stem cells
    • Ji, J., Ng, S. H., Sharma, V., Neculai, D., Hussein, S., Sam, M., Trinh, Q., Church, G. M., McPherson, J. D., Nagy, A. et al. (2012). Elevated coding mutation rate during the reprogramming of human somatic cells into induced pluripotent stem cells. Stem Cells 30, 435-440.
    • (2012) Stem Cells , vol.30 , pp. 435-440
    • Ji, J.1    Ng, S.H.2    Sharma, V.3    Neculai, D.4    Hussein, S.5    Sam, M.6    Trinh, Q.7    Church, G.M.8    McPherson, J.D.9    Nagy, A.10
  • 33
    • 83255177149 scopus 로고    scopus 로고
    • Donor cell type can influence the epigenome and differentiation potential of human induced pluripotent stem cells
    • Kim, K., Zhao, R., Doi, A., Ng, K., Unternaehrer, J., Cahan, P., Huo, H., Loh, Y. H., Aryee, M. J., Lensch, M. W. et al. (2011). Donor cell type can influence the epigenome and differentiation potential of human induced pluripotent stem cells. Nat. Biotechnol. 29, 1117-1119.
    • (2011) Nat. Biotechnol. , vol.29 , pp. 1117-1119
    • Kim, K.1    Zhao, R.2    Doi, A.3    Ng, K.4    Unternaehrer, J.5    Cahan, P.6    Huo, H.7    Loh, Y.H.8    Aryee, M.J.9    Lensch, M.W.10
  • 34
    • 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. (2011). 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 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
  • 36
    • 77954060569 scopus 로고    scopus 로고
    • Derivation of neural crest cells from human pluripotent stem cells
    • Lee, G., Chambers, S. M., Tomishima, M. J. and Studer, L. (2010). Derivation of neural crest cells from human pluripotent stem cells. Nat. Protoc. 5, 688-701.
    • (2010) Nat. Protoc. , vol.5 , pp. 688-701
    • Lee, G.1    Chambers, S.M.2    Tomishima, M.J.3    Studer, L.4
  • 37
    • 82655168715 scopus 로고    scopus 로고
    • Efficient correction of hemoglobinopathy-causing mutations by homologous recombination in integration-free patient iPSCs
    • Li, M., Suzuki, K., Qu, J., Saini, P., Dubova, I., Yi, F., Lee, J., Sancho-Martinez, I., Liu, G. H. and Izpisua Belmonte, J. C. (2011). Efficient correction of hemoglobinopathy-causing mutations by homologous recombination in integration-free patient iPSCs. Cell Res. 21, 1740-1744.
    • (2011) Cell Res. , vol.21 , pp. 1740-1744
    • Li, M.1    Suzuki, K.2    Qu, J.3    Saini, P.4    Dubova, I.5    Yi, F.6    Lee, J.7    Sancho-Martinez, I.8    Liu, G.H.9    Izpisua Belmonte, J.C.10
  • 38
    • 84872016692 scopus 로고    scopus 로고
    • Directed cardiomyocyte differentiation from human pluripotent stem cells by modulating Wnt/β-catenin signaling under fully defined conditions
    • Lian, X., Zhang, J., Azarin, S. M., Zhu, K., Hazeltine, L. B., Bao, X., Hsiao, C., Kamp, T. J. and Palecek, S. P. (2013). Directed cardiomyocyte differentiation from human pluripotent stem cells by modulating Wnt/β-catenin signaling under fully defined conditions. Nat. Protoc. 8, 162-175.
    • (2013) Nat. Protoc. , vol.8 , pp. 162-175
    • Lian, X.1    Zhang, J.2    Azarin, S.M.3    Zhu, K.4    Hazeltine, L.B.5    Bao, X.6    Hsiao, C.7    Kamp, T.J.8    Palecek, S.P.9
  • 45
    • 70349146995 scopus 로고    scopus 로고
    • Oligomerized pool engineering (OPEN): An 'open-source' protocol for making customized zinc-finger arrays
    • Maeder, M. L., Thibodeau-Beganny, S., Sander, J. D., Voytas, D. F. and Joung, J. K. (2009). Oligomerized pool engineering (OPEN): an 'open-source' protocol for making customized zinc-finger arrays. Nat. Protoc. 4, 1471-1501.
    • (2009) Nat. Protoc. , vol.4 , pp. 1471-1501
    • Maeder, M.L.1    Thibodeau-Beganny, S.2    Sander, J.D.3    Voytas, D.F.4    Joung, J.K.5
  • 48
    • 78149488365 scopus 로고    scopus 로고
    • A model for neural development and treatment of Rett syndrome using human induced pluripotent stem cells
    • Marchetto, M. C., Carromeu, C., Acab, A., Yu, D., Yeo, G. W., Mu, Y., Chen, G., Gage, F. H. and Muotri, A. R. (2010). A model for neural development and treatment of Rett syndrome using human induced pluripotent stem cells. Cell 143, 527-539.
    • (2010) Cell , vol.143 , pp. 527-539
    • Marchetto, M.C.1    Carromeu, C.2    Acab, A.3    Yu, D.4    Yeo, G.W.5    Mu, Y.6    Chen, G.7    Gage, F.H.8    Muotri, A.R.9
  • 54
    • 72149090954 scopus 로고    scopus 로고
    • A simple cipher governs DNA recognition by TAL effectors
    • Moscou, M. J. and Bogdanove, A. J. (2009). A simple cipher governs DNA recognition by TAL effectors. Science 326, 1501.
    • (2009) Science , vol.326 , pp. 1501
    • Moscou, M.J.1    Bogdanove, A.J.2
  • 60
    • 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. and Liu, D. R. (2011). Revealing off-target cleavage specificities of zinc-finger nucleases by in vitro selection. Nat. Methods 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
  • 63
    • 17844387370 scopus 로고    scopus 로고
    • A computational study of off-target effects of RNA interference
    • DOI 10.1093/nar/gki324
    • Qiu, S., Adema, C. M. and Lane, T. (2005). A computational study of off-target effects of RNA interference. Nucleic Acids Res. 33, 1834-1847. (Pubitemid 41748330)
    • (2005) Nucleic Acids Research , vol.33 , Issue.6 , pp. 1834-1847
    • Qiu, S.1    Adema, C.M.2    Lane, T.3
  • 67
    • 0034101804 scopus 로고    scopus 로고
    • Embryonic stem cell lines from human blastocysts: Somatic differentiation in vitro
    • DOI 10.1038/74447
    • Reubinoff, B. E., Pera, M. F., Fong, C. Y., Trounson, A. and Bongso, A. (2000). Embryonic stem cell lines from human blastocysts: somatic differentiation in vitro. Nat. Biotechnol. 18, 399-404. (Pubitemid 30217526)
    • (2000) Nature Biotechnology , vol.18 , Issue.4 , pp. 399-404
    • Reubinoff, B.E.1    Pera, M.F.2    Fong, C.-Y.3    Trounson, A.4    Bongso, A.5
  • 70
    • 0031897270 scopus 로고    scopus 로고
    • Human gene targeting by viral vectors
    • DOI 10.1038/ng0498-325
    • Russell, D. W. and Hirata, R. K. (1998). Human gene targeting by viral vectors. Nat. Genet. 18, 325-330. (Pubitemid 28158159)
    • (1998) Nature Genetics , vol.18 , Issue.4 , pp. 325-330
    • Russell, D.W.1    Hirata, R.K.2
  • 72
    • 84861170955 scopus 로고    scopus 로고
    • A transcription activator-like effector toolbox for genome engineering
    • Sanjana, N. E., Cong, L., Zhou, Y., Cunniff, M. M., Feng, G. and Zhang, F. (2012). A transcription activator-like effector toolbox for genome engineering. Nat. Protoc. 7, 171-192.
    • (2012) Nat. Protoc. , vol.7 , pp. 171-192
    • Sanjana, N.E.1    Cong, L.2    Zhou, Y.3    Cunniff, M.M.4    Feng, G.5    Zhang, F.6
  • 73
  • 75
    • 0022389290 scopus 로고
    • Insertion of DNA sequences into the human chromosomal β-globin locus by homologous recombination
    • DOI 10.1038/317230a0
    • Smithies, O., Gregg, R. G., Boggs, S. S., Koralewski, M. A. and Kucherlapati, R. S. (1985). Insertion of DNA sequences into the human chromosomal β-globin locus by homologous recombination. Nature 317, 230-234. (Pubitemid 16246494)
    • (1985) Nature , vol.317 , Issue.6034 , pp. 230-234
    • Smithies, O.1    Gregg, R.G.2    Boggs, S.S.3
  • 80
    • 78149302874 scopus 로고    scopus 로고
    • Efficient generation of functional dopaminergic neurons from human induced pluripotent stem cells under defined conditions
    • Swistowski, A., Peng, J., Liu, Q., Mali, P., Rao, M. S., Cheng, L. and Zeng, X. (2010). Efficient generation of functional dopaminergic neurons from human induced pluripotent stem cells under defined conditions. Stem Cells 28, 1893-1904.
    • (2010) Stem Cells , vol.28 , pp. 1893-1904
    • Swistowski, A.1    Peng, J.2    Liu, Q.3    Mali, P.4    Rao, M.S.5    Cheng, L.6    Zeng, X.7
  • 83
    • 36248966518 scopus 로고    scopus 로고
    • Induction of pluripotent stem cells from adult human fibroblasts by defined factors
    • Takahashi, K., Tanabe, K., Ohnuki, M., Narita, M., Ichisaka, T., Tomoda, K. and Yamanaka, S. (2007). Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell 131, 861-872.
    • (2007) Cell , vol.131 , pp. 861-872
    • Takahashi, K.1    Tanabe, K.2    Ohnuki, M.3    Narita, M.4    Ichisaka, T.5    Tomoda, K.6    Yamanaka, S.7
  • 84
    • 0023646810 scopus 로고
    • Site-directed mutagenesis by gene targeting in mouse embryo-derived stem cells
    • Thomas, K. R. and Capecchi, M. R. (1987). Site-directed mutagenesis by gene targeting in mouse embryo-derived stem cells. Cell 51, 503-512.
    • (1987) Cell , vol.51 , pp. 503-512
    • Thomas, K.R.1    Capecchi, M.R.2
  • 85
    • 0032491416 scopus 로고    scopus 로고
    • Embryonic stem cell lines derived from human blastocysts
    • Thomson, J. A., Itskovitz-Eldor, J., Shapiro, S. S., Waknitz, M. A., Swiergiel, J. J., Marshall, V. S. and Jones, J. M. (1998). Embryonic stem cell lines derived from human blastocysts. Science 282, 1145-1147. (Pubitemid 28516247)
    • (1998) Science , vol.282 , Issue.5391 , pp. 1145-1147
    • Thomson, J.A.1
  • 87
    • 70450247528 scopus 로고    scopus 로고
    • Human-induced pluripotent stem cells from blood cells of healthy donors and patients with acquired blood disorders
    • Ye, Z., Zhan, H., Mali, P., Dowey, S., Williams, D. M., Jang, Y. Y., Dang, C. V., Spivak, J. L., Moliterno, A. R. and Cheng, L. (2009). Human-induced pluripotent stem cells from blood cells of healthy donors and patients with acquired blood disorders. Blood 114, 5473-5480.
    • (2009) Blood , vol.114 , pp. 5473-5480
    • Ye, Z.1    Zhan, H.2    Mali, P.3    Dowey, S.4    Williams, D.M.5    Jang, Y.Y.6    Dang, C.V.7    Spivak, J.L.8    Moliterno, A.R.9    Cheng, L.10
  • 90
    • 79751487297 scopus 로고    scopus 로고
    • Efficient construction of sequence-specific TAL effectors for modulating mammalian transcription
    • Zhang, F., Cong, L., Lodato, S., Kosuri, S., Church, G. M. and Arlotta, P. (2011). Efficient construction of sequence-specific TAL effectors for modulating mammalian transcription. Nat. Biotechnol. 29, 149-153.
    • (2011) Nat. Biotechnol. , vol.29 , pp. 149-153
    • Zhang, F.1    Cong, L.2    Lodato, S.3    Kosuri, S.4    Church, G.M.5    Arlotta, P.6
  • 92
    • 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. and Cheng, L. (2011). Site-specific gene correction of a point mutation in human iPS cells derived from an adult patient with sickle cell disease. Blood 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
  • 93
    • 0037336820 scopus 로고    scopus 로고
    • Homologous recombination in human embryonic stem cells
    • DOI 10.1038/nbt788
    • Zwaka, T. P. and Thomson, J. A. (2003). Homologous recombination in human embryonic stem cells. Nat. Biotechnol. 21, 319-321. (Pubitemid 36314818)
    • (2003) Nature Biotechnology , vol.21 , Issue.3 , pp. 319-321
    • Zwaka, T.P.1    Thomson, J.A.2


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.