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Volumn 5, Issue , 2015, Pages

Both TALENs and CRISPR/Cas9 directly target the HBB IVS2-654 (C > T) mutation in β-thalassemiaderived iPSCs

Author keywords

[No Author keywords available]

Indexed keywords

HEMOGLOBIN BETA CHAIN;

EID: 84937019827     PISSN: None     EISSN: 20452322     Source Type: Journal    
DOI: 10.1038/srep12065     Document Type: Article
Times cited : (133)

References (49)
  • 2
    • 84889000317 scopus 로고    scopus 로고
    • Transcription activator-like effector nuclease (TALEN)-mediated gene correction in integration-free beta-thalassemia induced pluripotent stem cells
    • Ma, N. et al. Transcription activator-like effector nuclease (TALEN)-mediated gene correction in integration-free beta-thalassemia induced pluripotent stem cells. J Biol Chem 288, 34671-34679 (2013).
    • (2013) J Biol Chem , vol.288 , pp. 34671-34679
    • Ma, N.1
  • 3
    • 0035320886 scopus 로고    scopus 로고
    • Phenotype-genotype relationships in monogenic disease: Lessons from the thalassaemias
    • Weatherall, D. J. Phenotype-genotype relationships in monogenic disease: lessons from the thalassaemias. Nat Rev Genet 2, 245-255 (2001).
    • (2001) Nat Rev Genet , vol.2 , pp. 245-255
    • Weatherall, D.J.1
  • 4
    • 84871907955 scopus 로고    scopus 로고
    • Hematopoietic stem cell transplantation in thalassemia and sickle cell disease. Unicenter experience in a multi-ethnic population
    • Marziali, M., Isgro, A., Gaziev, J. & Lucarelli, G. Hematopoietic stem cell transplantation in thalassemia and sickle cell disease. Unicenter experience in a multi-ethnic population. Mediterr J Hematol Infect Dis 1, e2009027 (2009).
    • (2009) Mediterr J Hematol Infect Dis , vol.1 , pp. e2009027
    • Marziali, M.1    Isgro, A.2    Gaziev, J.3    Lucarelli, G.4
  • 5
    • 50949133924 scopus 로고    scopus 로고
    • Decreased differentiation of erythroid cells exacerbates ineffective erythropoiesis in beta-thalassemia
    • Libani, I. V. et al. Decreased differentiation of erythroid cells exacerbates ineffective erythropoiesis in beta-thalassemia. Blood 112, 875-885 (2008).
    • (2008) Blood , vol.112 , pp. 875-885
    • Libani, I.V.1
  • 7
    • 0023790820 scopus 로고
    • Molecular basis and prenatal diagnosis of beta-thalassemia
    • Kazazian, H. H., Jr. & Boehm, C. D. Molecular basis and prenatal diagnosis of beta-thalassemia. Blood 72, 1107-1116 (1988).
    • (1988) Blood , vol.72 , pp. 1107-1116
    • Kazazian, H.H.1    Boehm, C.D.2
  • 8
    • 0012401017 scopus 로고
    • Beta-Thalassemia in Chinese: Use of in vivo RNA analysis and oligonucleotide hybridization in systematic characterization of molecular defects
    • Cheng, T. C. et al. beta-Thalassemia in Chinese: use of in vivo RNA analysis and oligonucleotide hybridization in systematic characterization of molecular defects. Proc Natl Acad Sci USA 81, 2821-2825 (1984).
    • (1984) Proc Natl Acad Sci USA , vol.81 , pp. 2821-2825
    • Cheng, T.C.1
  • 9
    • 0023866477 scopus 로고
    • Molecular basis of beta thalassemia in south China. Strategy for DNA analysis
    • Zhang, J. Z. et al. Molecular basis of beta thalassemia in south China. Strategy for DNA analysis. Hum Genet 78, 37-40 (1988).
    • (1988) Hum Genet , vol.78 , pp. 37-40
    • Zhang, J.Z.1
  • 10
    • 0033558264 scopus 로고    scopus 로고
    • Bone marrow transplantation in adult thalassemic patients
    • Lucarelli, G. et al. Bone marrow transplantation in adult thalassemic patients. Blood 93, 1164-1167 (1999).
    • (1999) Blood , vol.93 , pp. 1164-1167
    • Lucarelli, G.1
  • 11
    • 77956928344 scopus 로고    scopus 로고
    • Transfusion independence and HMGA2 activation after gene therapy of human beta-thalassaemia
    • Cavazzana-Calvo, M. et al. Transfusion independence and HMGA2 activation after gene therapy of human beta-thalassaemia. Nature 467, 318-322 (2010).
    • (2010) Nature , vol.467 , pp. 318-322
    • Cavazzana-Calvo, M.1
  • 12
    • 0035861452 scopus 로고    scopus 로고
    • Correction of sickle cell disease in transgenic mouse models by gene therapy
    • Pawliuk, R. et al. Correction of sickle cell disease in transgenic mouse models by gene therapy. Science 294, 2368-2371 (2001).
    • (2001) Science , vol.294 , pp. 2368-2371
    • Pawliuk, R.1
  • 13
    • 69949150695 scopus 로고    scopus 로고
    • Generation of induced pluripotent stem cells from human beta-thalassemia fibroblast cells
    • Wang, Y., Jiang, Y., Liu, S., Sun, X. & Gao, S. Generation of induced pluripotent stem cells from human beta-thalassemia fibroblast cells. Cell Res 19, 1120-1123 (2009).
    • (2009) Cell Res , vol.19 , pp. 1120-1123
    • Wang, Y.1    Jiang, Y.2    Liu, S.3    Sun, X.4    Gao, S.5
  • 14
    • 84862776968 scopus 로고    scopus 로고
    • Genetic correction of beta-thalassemia patient-specific iPS cells and its use in improving hemoglobin production in irradiated SCID mice
    • Wang, Y. et al. Genetic correction of beta-thalassemia patient-specific iPS cells and its use in improving hemoglobin production in irradiated SCID mice. Cell Res 22, 637-648 (2012).
    • (2012) Cell Res , vol.22 , pp. 637-648
    • Wang, Y.1
  • 15
    • 70249114821 scopus 로고    scopus 로고
    • Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases
    • Hockemeyer, D. et al. Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases. Nat Biotechnol 27, 851-857 (2009).
    • (2009) Nat Biotechnol , vol.27 , pp. 851-857
    • Hockemeyer, D.1
  • 16
    • 79960836456 scopus 로고    scopus 로고
    • Genetic engineering of human pluripotent cells using TALE nucleases
    • Hockemeyer, D. et al. Genetic engineering of human pluripotent cells using TALE nucleases. Nat Biotechnol 29, 731-734 (2011).
    • (2011) Nat Biotechnol , vol.29 , pp. 731-734
    • Hockemeyer, D.1
  • 17
    • 84873729095 scopus 로고    scopus 로고
    • Multiplex genome engineering using CRISPR/Cas systems
    • Cong, L. et al. Multiplex genome engineering using CRISPR/Cas systems. Science 339, 819-823 (2013).
    • (2013) Science , vol.339 , pp. 819-823
    • Cong, L.1
  • 18
    • 84873734105 scopus 로고    scopus 로고
    • RNA-guided human genome engineering via Cas9
    • Mali, P. et al. RNA-guided human genome engineering via Cas9. Science 339, 823-826 (2013).
    • (2013) Science , vol.339 , pp. 823-826
    • Mali, P.1
  • 19
    • 84868608889 scopus 로고    scopus 로고
    • Zinc-finger nuclease-mediated correction of alpha-thalassemia in iPS cells
    • Chang, C. J. & Bouhassira, E. E. Zinc-finger nuclease-mediated correction of alpha-thalassemia in iPS cells. Blood 120, 3906- 3914 (2012).
    • (2012) Blood , vol.120 , pp. 3906-3914
    • Chang, C.J.1    Bouhassira, E.E.2
  • 20
    • 42949083192 scopus 로고    scopus 로고
    • Unexpected failure rates for modular assembly of engineered zinc fingers
    • Ramirez, C. L. et al. Unexpected failure rates for modular assembly of engineered zinc fingers. Nat Methods 5, 374-375 (2008).
    • (2008) Nat Methods , vol.5 , pp. 374-375
    • Ramirez, C.L.1
  • 21
    • 84907219050 scopus 로고    scopus 로고
    • Seamless gene correction of beta-thalassemia mutations in patient-specific iPSCs using CRISPR/Cas9 and piggyBac
    • Xie, F. et al. Seamless gene correction of beta-thalassemia mutations in patient-specific iPSCs using CRISPR/Cas9 and piggyBac. Genome Res 24, 1526-1533 (2014).
    • (2014) Genome Res , vol.24 , pp. 1526-1533
    • Xie, F.1
  • 22
    • 84934285785 scopus 로고    scopus 로고
    • Improved hematopoietic differentiation efficiency of gene-corrected beta-thalassemia induced pluripotent stem cells by CRISPR/Cas9 system
    • Song, B. et al. Improved Hematopoietic Differentiation Efficiency of Gene-Corrected Beta-Thalassemia Induced Pluripotent Stem Cells by CRISPR/Cas9 System. Stem Cells Dev 24, 1053-1065 (2015).
    • (2015) Stem Cells Dev , vol.24 , pp. 1053-1065
    • Song, B.1
  • 23
    • 0033762448 scopus 로고    scopus 로고
    • A new positive/negative selectable marker, puDeltatk, for use in embryonic stem cells
    • Chen, Y. T. & Bradley, A. A new positive/negative selectable marker, puDeltatk, for use in embryonic stem cells. Genesis 28, 31-35 (2000).
    • (2000) Genesis , vol.28 , pp. 31-35
    • Chen, Y.T.1    Bradley, A.2
  • 24
    • 84864475122 scopus 로고    scopus 로고
    • TAL Effector-Nucleotide Targeter (TALE-NT) 2.0: Tools for TAL effector design and target prediction
    • Doyle, E. L. et al. TAL Effector-Nucleotide Targeter (TALE-NT) 2.0: tools for TAL effector design and target prediction. Nucleic Acids Res 40, W117-W122 (2012).
    • (2012) Nucleic Acids Res , vol.40 , pp. W117-W122
    • Doyle, E.L.1
  • 25
    • 84896308706 scopus 로고    scopus 로고
    • Cas-OFFinder: A fast and versatile algorithm that searches for potential off-target sites of Cas9 RNAguided endonucleases
    • Bae, S., Park, J. & Kim, J. S. Cas-OFFinder: a fast and versatile algorithm that searches for potential off-target sites of Cas9 RNAguided endonucleases. Bioinformatics 30, 1473-1475 (2014).
    • (2014) Bioinformatics , vol.30 , pp. 1473-1475
    • Bae, S.1    Park, J.2    Kim, J.S.3
  • 26
    • 0023043553 scopus 로고
    • Introduction of homologous DNA sequences into mammalian cells induces mutations in the cognate gene
    • Thomas, K. R. & Capecchi, M. R. Introduction of homologous DNA sequences into mammalian cells induces mutations in the cognate gene. Nature 324, 34-38 (1986).
    • (1986) Nature , vol.324 , pp. 34-38
    • Thomas, K.R.1    Capecchi, M.R.2
  • 27
    • 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 317, 230-234 (1985).
    • (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
  • 28
    • 0022461705 scopus 로고
    • Germ-line transmission of genes introduced into cultured pluripotential cells by retroviral vector
    • Robertson, E., Bradley, A., Kuehn, M. & Evans, M. Germ-line transmission of genes introduced into cultured pluripotential cells by retroviral vector. Nature 323, 445-448 (1986).
    • (1986) Nature , vol.323 , pp. 445-448
    • Robertson, E.1    Bradley, A.2    Kuehn, M.3    Evans, M.4
  • 29
    • 47349097567 scopus 로고    scopus 로고
    • Rapid "open-source" engineering of customized zinc-finger nucleases for highly efficient gene modification
    • Maeder, M. L. et al. Rapid "open-source" engineering of customized zinc-finger nucleases for highly efficient gene modification. Mol Cell 31, 294-301 (2008).
    • (2008) Mol Cell , vol.31 , pp. 294-301
    • Maeder, M.L.1
  • 30
    • 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 16, 707-717 (2008).
    • (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
    • 84875157258 scopus 로고    scopus 로고
    • A library of TAL effector nucleases spanning the human genome
    • Kim, Y. et al. A library of TAL effector nucleases spanning the human genome. Nat Biotechnol 31, 251-258 (2013).
    • (2013) Nat Biotechnol , vol.31 , pp. 251-258
    • Kim, Y.1
  • 32
    • 84924033062 scopus 로고    scopus 로고
    • Efficient and allele-specific genome editing of disease loci in human iPSCs
    • Smith, C. et al. Efficient and allele-specific genome editing of disease loci in human iPSCs. Mol Ther 23, 570-577 (2015).
    • (2015) Mol Ther , vol.23 , pp. 570-577
    • Smith, C.1
  • 33
    • 84884950106 scopus 로고    scopus 로고
    • CRISPR/Cas9 systems targeting beta-globin and CCR5 genes have substantial off-target activity
    • Cradick, T. J., Fine, E. J., Antico, C. J. & Bao, G. CRISPR/Cas9 systems targeting beta-globin and CCR5 genes have substantial off-target activity. Nucleic Acids Res 41, 9584-9592 (2013).
    • (2013) Nucleic Acids Res , vol.41 , pp. 9584-9592
    • Cradick, T.J.1    Fine, E.J.2    Antico, C.J.3    Bao, G.4
  • 34
    • 84875963894 scopus 로고    scopus 로고
    • Enhanced efficiency of human pluripotent stem cell genome editing through replacing TALENs with CRISPRs
    • Ding, Q. et al. Enhanced efficiency of human pluripotent stem cell genome editing through replacing TALENs with CRISPRs. Cell Stem Cell 12, 393-394 (2013).
    • (2013) Cell Stem Cell , vol.12 , pp. 393-394
    • Ding, Q.1
  • 35
    • 84884288934 scopus 로고    scopus 로고
    • Double nicking by RNA-guided CRISPR Cas9 for enhanced genome editing specificity
    • Ran, F. A. et al. Double nicking by RNA-guided CRISPR Cas9 for enhanced genome editing specificity. Cell 154, 1380-1389 (2013).
    • (2013) Cell , vol.154 , pp. 1380-1389
    • Ran, F.A.1
  • 36
    • 84874613680 scopus 로고    scopus 로고
    • Obligate ligation-gated recombination (ObLiGaRe): Custom-designed nucleasemediated targeted integration through nonhomologous end joining
    • Maresca, M., Lin, V. G., Guo, N. & Yang, Y. Obligate ligation-gated recombination (ObLiGaRe): custom-designed nucleasemediated targeted integration through nonhomologous end joining. Genome Res 23, 539-546 (2013).
    • (2013) Genome Res , vol.23 , pp. 539-546
    • Maresca, M.1    Lin, V.G.2    Guo, N.3    Yang, Y.4
  • 37
    • 84865070369 scopus 로고    scopus 로고
    • A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity
    • Jinek, M. et al. A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity. Science 337, 816-821 (2012).
    • (2012) Science , vol.337 , pp. 816-821
    • Jinek, M.1
  • 38
    • 84896267457 scopus 로고    scopus 로고
    • Homology-directed repair of DNA nicks via pathways distinct from canonical double-strand break repair
    • Davis, L. & Maizels, N. Homology-directed repair of DNA nicks via pathways distinct from canonical double-strand break repair. Proc Natl Acad Sci USA 111, E924-E932 (2014).
    • (2014) Proc Natl Acad Sci USA , vol.111 , pp. E924-E932
    • Davis, L.1    Maizels, N.2
  • 39
    • 84884160273 scopus 로고    scopus 로고
    • CAS9 transcriptional activators for target specificity screening and paired nickases for cooperative genome engineering
    • Mali, P. et al. CAS9 transcriptional activators for target specificity screening and paired nickases for cooperative genome engineering. Nat Biotechnol 31, 833-838 (2013).
    • (2013) Nat Biotechnol , vol.31 , pp. 833-838
    • Mali, P.1
  • 40
    • 84923081561 scopus 로고    scopus 로고
    • Genome-wide detection of DNA double-stranded breaks induced by engineered nucleases
    • Frock, R. L. et al. Genome-wide detection of DNA double-stranded breaks induced by engineered nucleases. Nat Biotechnol (2014).
    • (2014) Nat Biotechnol
    • Frock, R.L.1
  • 41
    • 84903545084 scopus 로고    scopus 로고
    • Genome-wide analysis reveals characteristics of off-target sites bound by the Cas9 endonuclease
    • Kuscu, C., Arslan, S., Singh, R., Thorpe, J. & Adli, M. Genome-wide analysis reveals characteristics of off-target sites bound by the Cas9 endonuclease. Nat Biotechnol 32, 677-683 (2014).
    • (2014) Nat Biotechnol , vol.32 , pp. 677-683
    • Kuscu, C.1    Arslan, S.2    Singh, R.3    Thorpe, J.4    Adli, M.5
  • 42
    • 84923221641 scopus 로고    scopus 로고
    • Unbiased detection of off-target cleavage by CRISPR-Cas9 and TALENs using integrase-defective lentiviral vectors
    • Wang, X. et al. Unbiased detection of off-target cleavage by CRISPR-Cas9 and TALENs using integrase-defective lentiviral vectors. Nat Biotechnol (2015).
    • (2015) Nat Biotechnol
    • Wang, X.1
  • 43
    • 84903943282 scopus 로고    scopus 로고
    • Whole-genome sequencing analysis reveals high specificity of CRISPR/Cas9 and TALEN-based genome editing in human iPSCs
    • Smith, C. et al. Whole-genome sequencing analysis reveals high specificity of CRISPR/Cas9 and TALEN-based genome editing in human iPSCs. Cell Stem Cell 15, 12-13 (2014).
    • (2014) Cell Stem Cell , vol.15 , pp. 12-13
    • Smith, C.1
  • 44
    • 84904010334 scopus 로고    scopus 로고
    • Low incidence of off-target mutations in individual CRISPR-Cas9 and TALEN targeted human stem cell clones detected by whole-genome sequencing
    • Veres, A. et al. Low incidence of off-target mutations in individual CRISPR-Cas9 and TALEN targeted human stem cell clones detected by whole-genome sequencing. Cell Stem Cell 15, 27-30 (2014).
    • (2014) Cell Stem Cell , vol.15 , pp. 27-30
    • Veres, A.1
  • 45
    • 84891710947 scopus 로고    scopus 로고
    • Analysis of off-target effects of CRISPR/Cas-derived RNA-guided endonucleases and nickases
    • Cho, S. W. et al. Analysis of off-target effects of CRISPR/Cas-derived RNA-guided endonucleases and nickases. Genome Res 24, 132-141 (2014).
    • (2014) Genome Res , vol.24 , pp. 132-141
    • Cho, S.W.1
  • 46
    • 84897954175 scopus 로고    scopus 로고
    • Efficient genome modification by CRISPR-Cas9 nickase with minimal off-target effects
    • Shen, B. et al. Efficient genome modification by CRISPR-Cas9 nickase with minimal off-target effects. Nat Methods 11, 399-402 (2014).
    • (2014) Nat Methods , vol.11 , pp. 399-402
    • Shen, B.1
  • 47
    • 84923221641 scopus 로고    scopus 로고
    • Unbiased detection of off-target cleavage by CRISPR-Cas9 and TALENs using integrase-defective lentiviral vectors
    • Wang, X. et al. Unbiased detection of off-target cleavage by CRISPR-Cas9 and TALENs using integrase-defective lentiviral vectors. Nat Biotechnol 33, 175-178 (2015).
    • (2015) Nat Biotechnol , vol.33 , pp. 175-178
    • Wang, X.1
  • 48
    • 80054987997 scopus 로고    scopus 로고
    • Targeted gene correction of alpha1-antitrypsin deficiency in induced pluripotent stem cells
    • Yusa, K. et al. Targeted gene correction of alpha1-antitrypsin deficiency in induced pluripotent stem cells. Nature 478, 391-394 (2011).
    • (2011) Nature , vol.478 , pp. 391-394
    • Yusa, K.1
  • 49
    • 84897042820 scopus 로고    scopus 로고
    • Seamless correction of the sickle cell disease mutation of the HBB gene in human induced pluripotent stem cells using TALENs
    • Sun, N. & Zhao, H. Seamless correction of the sickle cell disease mutation of the HBB gene in human induced pluripotent stem cells using TALENs. Biotechnol Bioeng 111, 1048-1053 (2014).
    • (2014) Biotechnol Bioeng , vol.111 , pp. 1048-1053
    • Sun, N.1    Zhao, H.2


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