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Volumn 127, Issue 21, 2016, Pages 2546-2552

The clinical applications of genome editing in HIV

Author keywords

[No Author keywords available]

Indexed keywords

ANTIRETROVIRUS AGENT; CHEMOKINE RECEPTOR CCR5; ZINC FINGER NUCLEASE; CCR5 PROTEIN, HUMAN; DEOXYRIBONUCLEASE;

EID: 84974569561     PISSN: 00064971     EISSN: 15280020     Source Type: Journal    
DOI: 10.1182/blood-2016-01-678144     Document Type: Review
Times cited : (59)

References (90)
  • 1
    • 0030913366 scopus 로고    scopus 로고
    • Quantification of latent tissue reservoirs and total body viral load in HIV-1 infection
    • Chun TW, Carruth L, Finzi D, et al. Quantification of latent tissue reservoirs and total body viral load in HIV-1 infection. Nature. 1997;387(6629):183-188.
    • (1997) Nature , vol.387 , Issue.6629 , pp. 183-188
    • Chun, T.W.1    Carruth, L.2    Finzi, D.3
  • 2
    • 14444273439 scopus 로고    scopus 로고
    • Identification of a reservoir for HIV-1 in patients on highly active antiretroviral therapy
    • Finzi D, Hermankova M, Pierson T, et al. Identification of a reservoir for HIV-1 in patients on highly active antiretroviral therapy. Science. 1997;278(5341):1295-1300.
    • (1997) Science , vol.278 , Issue.5341 , pp. 1295-1300
    • Finzi, D.1    Hermankova, M.2    Pierson, T.3
  • 3
    • 0032953920 scopus 로고    scopus 로고
    • Latent infection of CD41 T cells provides a mechanism for lifelong persistence of HIV-1, even in patients on effective combination therapy
    • Finzi D, Blankson J, Siliciano JD, et al. Latent infection of CD41 T cells provides a mechanism for lifelong persistence of HIV-1, even in patients on effective combination therapy. Nat Med. 1999;5(5):512-517.
    • (1999) Nat Med , vol.5 , Issue.5 , pp. 512-517
    • Finzi, D.1    Blankson, J.2    Siliciano, J.D.3
  • 4
    • 0038579210 scopus 로고    scopus 로고
    • Long-term follow-up studies confirm the stability of the latent reservoir for HIV-1 in resting CD4+ T cells
    • Siliciano JD, Kajdas J, Finzi D, et al. Long-term follow-up studies confirm the stability of the latent reservoir for HIV-1 in resting CD4+ T cells. Nat Med. 2003;9(6):727-728.
    • (2003) Nat Med , vol.9 , Issue.6 , pp. 727-728
    • Siliciano, J.D.1    Kajdas, J.2    Finzi, D.3
  • 6
    • 62449222132 scopus 로고    scopus 로고
    • Incomplete peripheral CD41 cell count restoration in HIV-infected patients receiving long-term antiretroviral treatment
    • Kelley CF, Kitchen CM, Hunt PW, et al. Incomplete peripheral CD41 cell count restoration in HIV-infected patients receiving long-term antiretroviral treatment. Clin Infect Dis. 2009;48(6):787-794.
    • (2009) Clin Infect Dis , vol.48 , Issue.6 , pp. 787-794
    • Kelley, C.F.1    Kitchen, C.M.2    Hunt, P.W.3
  • 8
    • 84875959697 scopus 로고    scopus 로고
    • T-cell therapies for HIV
    • Lam S, Bollard C. T-cell therapies for HIV. Immunotherapy. 2013;5(4):407-414.
    • (2013) Immunotherapy , vol.5 , Issue.4 , pp. 407-414
    • Lam, S.1    Bollard, C.2
  • 9
  • 10
    • 24944441656 scopus 로고    scopus 로고
    • Preferential survival of CD4+ T lymphocytes engineered with anti-human immunodeficiency virus (HIV) genes in HIV-infected individuals
    • Morgan RA, Walker R, Carter CS, et al. Preferential survival of CD4+ T lymphocytes engineered with anti-human immunodeficiency virus (HIV) genes in HIV-infected individuals. Hum Gene Ther. 2005;16(9):1065-1074.
    • (2005) Hum Gene Ther , vol.16 , Issue.9 , pp. 1065-1074
    • Morgan, R.A.1    Walker, R.2    Carter, C.S.3
  • 11
    • 27744605569 scopus 로고    scopus 로고
    • Long-term inhibition of HIV-1 infection in primary hematopoietic cells by lentiviral vector delivery of a triple combination of anti-HIV shRNA, anti-CCR5 ribozyme, and a nucleolar-localizing TAR decoy
    • Li MJ, Kim J, Li S, et al. Long-term inhibition of HIV-1 infection in primary hematopoietic cells by lentiviral vector delivery of a triple combination of anti-HIV shRNA, anti-CCR5 ribozyme, and a nucleolar-localizing TAR decoy. Mol Ther. 2005;12(5):900-909.
    • (2005) Mol Ther , vol.12 , Issue.5 , pp. 900-909
    • Li, M.J.1    Kim, J.2    Li, S.3
  • 12
    • 20844440241 scopus 로고    scopus 로고
    • Selective survival of peripheral blood lymphocytes in children with HIV-1 following delivery of an anti-HIV gene to bone marrow CD34(1) cells
    • Podsakoff GM, Engel BC, Carbonaro DA, et al. Selective survival of peripheral blood lymphocytes in children with HIV-1 following delivery of an anti-HIV gene to bone marrow CD34(1) cells. Mol Ther. 2005;12(1):77-86.
    • (2005) Mol Ther , vol.12 , Issue.1 , pp. 77-86
    • Podsakoff, G.M.1    Engel, B.C.2    Carbonaro, D.A.3
  • 13
    • 33751251058 scopus 로고    scopus 로고
    • Gene transfer in humans using a conditionally replicating lentiviral vector
    • Levine BL, Humeau LM, Boyer J, et al. Gene transfer in humans using a conditionally replicating lentiviral vector. Proc Natl Acad Sci USA. 2006;103(46):17372-17377.
    • (2006) Proc Natl Acad Sci USA , vol.103 , Issue.46 , pp. 17372-17377
    • Levine, B.L.1    Humeau, L.M.2    Boyer, J.3
  • 14
    • 34247208235 scopus 로고    scopus 로고
    • Transfer of autologous gene-modified T cells in HIV-infected patients with advanced immunodeficiency and drug-resistant virus
    • van Lunzen J, Glaunsinger T, Stahmer I, et al. Transfer of autologous gene-modified T cells in HIV-infected patients with advanced immunodeficiency and drug-resistant virus. Mol Ther. 2007;15(5):1024-1033.
    • (2007) Mol Ther , vol.15 , Issue.5 , pp. 1024-1033
    • Van Lunzen, J.1    Glaunsinger, T.2    Stahmer, I.3
  • 15
    • 62049083710 scopus 로고    scopus 로고
    • Phase 2 gene therapy trial of an anti-HIV ribozyme in autologous CD341 cells
    • Mitsuyasu RT, Merigan TC, Carr A, et al. Phase 2 gene therapy trial of an anti-HIV ribozyme in autologous CD341 cells. Nat Med. 2009;15(3):285-292.
    • (2009) Nat Med , vol.15 , Issue.3 , pp. 285-292
    • Mitsuyasu, R.T.1    Merigan, T.C.2    Carr, A.3
  • 16
    • 77955608799 scopus 로고    scopus 로고
    • RNA-based gene therapy for HIV with lentiviral vectormodified CD34(+) cells in patients undergoing transplantation for AIDS-related lymphoma
    • DiGiusto DL, Krishnan A, Li L, et al. RNA-based gene therapy for HIV with lentiviral vectormodified CD34(+) cells in patients undergoing transplantation for AIDS-related lymphoma. Sci Transl Med. 2010;2(36):36ra43.
    • (2010) Sci Transl Med , vol.2 , Issue.36
    • DiGiusto, D.L.1    Krishnan, A.2    Li, L.3
  • 17
    • 33646887376 scopus 로고    scopus 로고
    • Genotoxicity of retroviral integration in hematopoietic cells
    • Nienhuis AW, Dunbar CE, Sorrentino BP. Genotoxicity of retroviral integration in hematopoietic cells. Mol Ther. 2006;13(6):1031-1049.
    • (2006) Mol Ther , vol.13 , Issue.6 , pp. 1031-1049
    • Nienhuis, A.W.1    Dunbar, C.E.2    Sorrentino, B.P.3
  • 18
    • 79953844921 scopus 로고    scopus 로고
    • Genotoxicity of retroviral hematopoietic stem cell gene therapy
    • Trobridge GD. Genotoxicity of retroviral hematopoietic stem cell gene therapy. Expert Opin Biol Ther. 2011;11(5):581-593.
    • (2011) Expert Opin Biol Ther , vol.11 , Issue.5 , pp. 581-593
    • Trobridge, G.D.1
  • 19
    • 0030812487 scopus 로고    scopus 로고
    • Homing endonucleases: Keeping the house in order
    • Belfort M, Roberts RJ. Homing endonucleases: keeping the house in order. Nucleic Acids Res. 1997;25(17):3379-3388.
    • (1997) Nucleic Acids Res , vol.25 , Issue.17 , pp. 3379-3388
    • Belfort, M.1    Roberts, R.J.2
  • 20
    • 84879264708 scopus 로고    scopus 로고
    • ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering
    • Gaj T, Gersbach CA, Barbas CF III. ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering. Trends Biotechnol. 2013;31(7):397-405.
    • (2013) Trends Biotechnol , vol.31 , Issue.7 , pp. 397-405
    • Gaj, T.1    Gersbach, C.A.2    Barbas, C.F.3
  • 21
    • 3142768860 scopus 로고    scopus 로고
    • Conservative homologous recombination preferentially repairs DNA double-strand breaks in the S phase of the cell cycle in human cells
    • Saleh-Gohari N, Helleday T. Conservative homologous recombination preferentially repairs DNA double-strand breaks in the S phase of the cell cycle in human cells. Nucleic Acids Res. 2004;32(12):3683-3688.
    • (2004) Nucleic Acids Res , vol.32 , Issue.12 , pp. 3683-3688
    • Saleh-Gohari, N.1    Helleday, T.2
  • 22
    • 84864321774 scopus 로고    scopus 로고
    • Quantitative live cell imaging reveals a gradual shift between DNA repair mechanisms and a maximal use of HR in mid S phase
    • Karanam K, Kafri R, Loewer A, Lahav G. Quantitative live cell imaging reveals a gradual shift between DNA repair mechanisms and a maximal use of HR in mid S phase. Mol Cell. 2012;47(2):320-329.
    • (2012) Mol Cell , vol.47 , Issue.2 , pp. 320-329
    • Karanam, K.1    Kafri, R.2    Loewer, A.3    Lahav, G.4
  • 23
    • 35548939825 scopus 로고    scopus 로고
    • The epidemiology of HIV coreceptor tropism
    • Hoffmann C. The epidemiology of HIV coreceptor tropism. Eur J Med Res. 2007;12(9):385-390.
    • (2007) Eur J Med Res , vol.12 , Issue.9 , pp. 385-390
    • Hoffmann, C.1
  • 24
    • 16044373004 scopus 로고    scopus 로고
    • Resistance to HIV-1 infection in caucasian individuals bearing mutant alleles of the CCR-5 chemokine receptor gene
    • Samson M, Libert F, Doranz BJ, et al. Resistance to HIV-1 infection in caucasian individuals bearing mutant alleles of the CCR-5 chemokine receptor gene. Nature. 1996;382(6593):722-725.
    • (1996) Nature , vol.382 , Issue.6593 , pp. 722-725
    • Samson, M.1    Libert, F.2    Doranz, B.J.3
  • 25
    • 16044367526 scopus 로고    scopus 로고
    • The role of a mutant CCR5 allele in HIV-1 transmission and disease progression
    • Huang Y, Paxton WA, Wolinsky SM, et al. The role of a mutant CCR5 allele in HIV-1 transmission and disease progression. Nat Med. 1996;2(11):1240-1243.
    • (1996) Nat Med , vol.2 , Issue.11 , pp. 1240-1243
    • Huang, Y.1    Paxton, W.A.2    Wolinsky, S.M.3
  • 26
    • 15844388931 scopus 로고    scopus 로고
    • Homozygous defect in HIV-1 coreceptor accounts for resistance of some multiply-exposed individuals to HIV-1 infection
    • Liu R, Paxton WA, Choe S, et al. Homozygous defect in HIV-1 coreceptor accounts for resistance of some multiply-exposed individuals to HIV-1 infection. Cell. 1996;86(3):367-377.
    • (1996) Cell , vol.86 , Issue.3 , pp. 367-377
    • Liu, R.1    Paxton, W.A.2    Choe, S.3
  • 27
    • 60549090253 scopus 로고    scopus 로고
    • Long-term control of HIV by CCR5 Delta32/Delta32 stem-cell transplantation
    • Hütter G, Nowak D, Mossner M, et al. Long-term control of HIV by CCR5 Delta32/Delta32 stem-cell transplantation. N Engl J Med. 2009;360(7):692-698.
    • (2009) N Engl J Med , vol.360 , Issue.7 , pp. 692-698
    • Hütter, G.1    Nowak, D.2    Mossner, M.3
  • 28
    • 79953117041 scopus 로고    scopus 로고
    • Evidence for the cure of HIV infection by CCR5Δ32/Δ32 stem cell transplantation
    • Allers K, Hütter G, Hofmann J, et al. Evidence for the cure of HIV infection by CCR5Δ32/Δ32 stem cell transplantation. Blood. 2011;117(10):2791-2799.
    • (2011) Blood , vol.117 , Issue.10 , pp. 2791-2799
    • Allers, K.1    Hütter, G.2    Hofmann, J.3
  • 29
    • 84877278315 scopus 로고    scopus 로고
    • Long-term reduction in peripheral blood HIV type 1 reservoirs following reduced-intensity conditioning allogeneic stem cell transplantation
    • Henrich TJ, Hu Z, Li JZ, et al. Long-term reduction in peripheral blood HIV type 1 reservoirs following reduced-intensity conditioning allogeneic stem cell transplantation. J Infect Dis. 2013;207(11):1694-1702.
    • (2013) J Infect Dis , vol.207 , Issue.11 , pp. 1694-1702
    • Henrich, T.J.1    Hu, Z.2    Li, J.Z.3
  • 30
    • 84900870642 scopus 로고    scopus 로고
    • HIV eradication-from Berlin to Boston
    • Cannon PM, Kohn DB, Kiem HP. HIV eradication-from Berlin to Boston. Nat Biotechnol. 2014;32(4):315-316.
    • (2014) Nat Biotechnol , vol.32 , Issue.4 , pp. 315-316
    • Cannon, P.M.1    Kohn, D.B.2    Kiem, H.P.3
  • 31
    • 79953123523 scopus 로고    scopus 로고
    • Chemokine receptor 5 knockout strategies
    • Cannon P, June C. Chemokine receptor 5 knockout strategies. Curr Opin HIV AIDS. 2011;6(1):74-79.
    • (2011) Curr Opin HIV AIDS , vol.6 , Issue.1 , pp. 74-79
    • Cannon, P.1    June, C.2
  • 32
    • 0030881882 scopus 로고    scopus 로고
    • Phenotypic knockout of HIV type 1 chemokine coreceptor CCR-5 by intrakines as potential therapeutic approach for HIV-1 infection
    • Yang AG, Bai X, Huang XF, Yao C, Chen S. Phenotypic knockout of HIV type 1 chemokine coreceptor CCR-5 by intrakines as potential therapeutic approach for HIV-1 infection. Proc Natl Acad Sci USA. 1997;94(21):11567-11572.
    • (1997) Proc Natl Acad Sci USA , vol.94 , Issue.21 , pp. 11567-11572
    • Yang, A.G.1    Bai, X.2    Huang, X.F.3    Yao, C.4    Chen, S.5
  • 33
    • 0034149743 scopus 로고    scopus 로고
    • Characterization of anti-CCR5 ribozyme-transduced CD34+ hematopoietic progenitor cells in vitro and in a SCID-hu mouse model in vivo
    • Bai J, Gorantla S, Banda N, Cagnon L, Rossi J, Akkina R. Characterization of anti-CCR5 ribozyme-transduced CD34+ hematopoietic progenitor cells in vitro and in a SCID-hu mouse model in vivo. Mol Ther. 2000;1(3):244-254.
    • (2000) Mol Ther , vol.1 , Issue.3 , pp. 244-254
    • Bai, J.1    Gorantla, S.2    Banda, N.3    Cagnon, L.4    Rossi, J.5    Akkina, R.6
  • 34
    • 8844259701 scopus 로고    scopus 로고
    • Protecting from R5-tropic HIV: Individual and combined effectiveness of a hammerhead ribozyme and a single-chain Fv antibody that targets CCR5
    • Cordelier P, Kulkowsky JW, Ko C, et al. Protecting from R5-tropic HIV: individual and combined effectiveness of a hammerhead ribozyme and a single-chain Fv antibody that targets CCR5. Gene Ther. 2004;11(22):1627-1637.
    • (2004) Gene Ther , vol.11 , Issue.22 , pp. 1627-1637
    • Cordelier, P.1    Kulkowsky, J.W.2    Ko, C.3
  • 35
    • 0037422583 scopus 로고    scopus 로고
    • Inhibiting HIV-1 infection in human T cells by lentiviralmediated delivery of small interfering RNA against CCR5
    • Qin XF, An DS,Chen IS, Baltimore D. Inhibiting HIV-1 infection in human T cells by lentiviralmediated delivery of small interfering RNA against CCR5. Proc Natl Acad Sci USA. 2003;100(1):183-188.
    • (2003) Proc Natl Acad Sci USA , vol.100 , Issue.1 , pp. 183-188
    • Qin, X.F.1    An, D.S.2    Chen, I.S.3    Baltimore, D.4
  • 36
    • 33845221484 scopus 로고    scopus 로고
    • HIV-1 resistance conferred by siRNA cosuppression of CXCR4 and CCR5 coreceptors by a bispecific lentiviral vector
    • Anderson J, Akkina R. HIV-1 resistance conferred by siRNA cosuppression of CXCR4 and CCR5 coreceptors by a bispecific lentiviral vector. AIDS Res Ther. 2005;2(1):1.
    • (2005) AIDS Res Ther , vol.2 , Issue.1 , pp. 1
    • Anderson, J.1    Akkina, R.2
  • 37
    • 84928948209 scopus 로고    scopus 로고
    • Genome editing technologies: Defining a path to clinic
    • Corrigan-Curay J, O'Reilly M, Kohn DB, et al. Genome editing technologies: defining a path to clinic. Mol Ther. 2015;23(5):796-806.
    • (2015) Mol Ther , vol.23 , Issue.5 , pp. 796-806
    • Corrigan-Curay, J.1    O'Reilly, M.2    Kohn, D.B.3
  • 38
    • 46949095221 scopus 로고    scopus 로고
    • Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases
    • Perez EE, Wang J, Miller JC, et al. Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases. Nat Biotechnol. 2008;26(7):808-816.
    • (2008) Nat Biotechnol , vol.26 , Issue.7 , pp. 808-816
    • Perez, E.E.1    Wang, J.2    Miller, J.C.3
  • 39
    • 78650735673 scopus 로고    scopus 로고
    • Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo
    • Holt N, Wang J, Kim K, et al. Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo. Nat Biotechnol. 2010;28(8):839-847.
    • (2010) Nat Biotechnol , vol.28 , Issue.8 , pp. 839-847
    • Holt, N.1    Wang, J.2    Kim, K.3
  • 40
    • 84875420616 scopus 로고    scopus 로고
    • Efficient clinical scale gene modification via zinc finger nuclease-targeted disruption of the HIV coreceptor CCR5
    • Maier DA, Brennan AL, Jiang S, et al. Efficient clinical scale gene modification via zinc finger nuclease-targeted disruption of the HIV coreceptor CCR5. Hum Gene Ther. 2013;24(3):245-258.
    • (2013) Hum Gene Ther , vol.24 , Issue.3 , pp. 245-258
    • Maier, D.A.1    Brennan, A.L.2    Jiang, S.3
  • 41
    • 84895487305 scopus 로고    scopus 로고
    • Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV
    • Tebas P, Stein D, Tang WW, et al. Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV. N Engl J Med. 2014;370(10):901-910.
    • (2014) N Engl J Med , vol.370 , Issue.10 , pp. 901-910
    • Tebas, P.1    Stein, D.2    Tang, W.W.3
  • 43
    • 84974531525 scopus 로고    scopus 로고
    • HIV protected autologous zinc finger nuclease CCR5 modified CD4 cells (SB-728-T) reduce Viral Load (VL) in HIV subjects during treatment interruption (TI): Correlates of effect, and effect of cytoxan conditioning
    • Accessed January 12, 2016
    • Ando D, Lalezari J, Blick G, et al. HIV protected autologous zinc finger nuclease CCR5 modified CD4 cells (SB-728-T) reduce Viral Load (VL) in HIV subjects during treatment interruption (TI): correlates of effect, and effect of cytoxan conditioning. ICAAC 2014. http://www.natap.org/2014/ICAAC/ICAAC-36.htm. Accessed January 12, 2016.
    • ICAAC 2014
    • Ando, D.1    Lalezari, J.2    Blick, G.3
  • 45
    • 84923266604 scopus 로고    scopus 로고
    • GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases
    • Tsai SQ, Zheng Z, Nguyen NT, et al. GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases. Nat Biotechnol. 2015;33(2):187-197.
    • (2015) Nat Biotechnol , vol.33 , Issue.2 , pp. 187-197
    • Tsai, S.Q.1    Zheng, Z.2    Nguyen, N.T.3
  • 46
    • 84923275611 scopus 로고    scopus 로고
    • Genome-wide detection of DNA double-stranded breaks induced by engineered nucleases
    • Frock RL, Hu J, Meyers RM, Ho YJ, Kii E, Alt FW. Genome-wide detection of DNA double-stranded breaks induced by engineered nucleases. Nat Biotechnol. 2015;33(2):179-186.
    • (2015) Nat Biotechnol , vol.33 , Issue.2 , pp. 179-186
    • Frock, R.L.1    Hu, J.2    Meyers, R.M.3    Ho, Y.J.4    Kii, E.5    Alt, F.W.6
  • 47
    • 0030860210 scopus 로고    scopus 로고
    • CCR2 chemokine receptor and AIDS progression
    • Smith MW, Carrington M, Winkler C, et al. CCR2 chemokine receptor and AIDS progression. Nat Med. 1997;3(10):1052-1053.
    • (1997) Nat Med , vol.3 , Issue.10 , pp. 1052-1053
    • Smith, M.W.1    Carrington, M.2    Winkler, C.3
  • 48
    • 84878528467 scopus 로고    scopus 로고
    • Genomic editing of the HIV-1 coreceptor CCR5 in adult hematopoietic stem and progenitor cells using zinc finger nucleases
    • Li L, Krymskaya L, Wang J, et al. Genomic editing of the HIV-1 coreceptor CCR5 in adult hematopoietic stem and progenitor cells using zinc finger nucleases. Mol Ther. 2013;21(6):1259-1269.
    • (2013) Mol Ther , vol.21 , Issue.6 , pp. 1259-1269
    • Li, L.1    Krymskaya, L.2    Wang, J.3
  • 49
    • 84949814888 scopus 로고    scopus 로고
    • Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors
    • Wang J, Exline CM, DeClercq JJ, et al. Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors. Nat Biotechnol. 2015;33(12):1256-1263.
    • (2015) Nat Biotechnol , vol.33 , Issue.12 , pp. 1256-1263
    • Wang, J.1    Exline, C.M.2    DeClercq, J.J.3
  • 50
    • 84890350615 scopus 로고    scopus 로고
    • Pre-clinical modeling of CCR5 knockout in human hematopoietic stem cells by zinc finger nucleases using humanized mice
    • Hofer U, Henley JE, Exline CM, Mulhern O, Lopez E, Cannon PM. Pre-clinical modeling of CCR5 knockout in human hematopoietic stem cells by zinc finger nucleases using humanized mice. J Infect Dis. 2013;208(Suppl 2):S160-S164.
    • (2013) J Infect Dis. , vol.208 , pp. S160-S164
    • Hofer, U.1    Henley, J.E.2    Exline, C.M.3    Mulhern, O.4    Lopez, E.5    Cannon, P.M.6
  • 51
    • 85002994386 scopus 로고    scopus 로고
    • Zinc finger nuclease editing of hematopoietic stem cells as an anti-HIV therapy
    • Poluektova LY, Garcia-Martinez JV, Koyanagi Y, Manz MG, Tager AM, eds. New York: Springer
    • Holt N, Exline CM, Mulhern O, et al. Zinc finger nuclease editing of hematopoietic stem cells as an anti-HIV therapy.In: Poluektova LY, Garcia-Martinez JV, Koyanagi Y, Manz MG, Tager AM, eds. Humanized Mice for HIV Research. New York: Springer; 2014:407-416.
    • (2014) Humanized Mice for HIV Research , pp. 407-416
    • Holt, N.1    Exline, C.M.2    Mulhern, O.3
  • 52
    • 85002949800 scopus 로고    scopus 로고
    • Electroporation of ZFN mRNA enables efficient CCR5 gene disruption in mobilized blood hematopoietic stem cells at clinical scale
    • Cannon PM, Henley J, Wood T, et al. Electroporation of ZFN mRNA enables efficient CCR5 gene disruption in mobilized blood hematopoietic stem cells at clinical scale. Mol Ther. 2013;21(S1):S71-S72.
    • (2013) Mol Ther , vol.21 , Issue.S1 , pp. S71-S72
    • Cannon, P.M.1    Henley, J.2    Wood, T.3
  • 53
    • 84902315464 scopus 로고    scopus 로고
    • Targeted genome editing in human repopulating haematopoietic stem cells
    • Genovese P, Schiroli G, Escobar G, et al. Targeted genome editing in human repopulating haematopoietic stem cells. Nature. 2014;510(7504):235-240.
    • (2014) Nature , vol.510 , Issue.7504 , pp. 235-240
    • Genovese, P.1    Schiroli, G.2    Escobar, G.3
  • 54
    • 65249102249 scopus 로고    scopus 로고
    • Hematopoietic and endothelial differentiation of human induced pluripotent stem cells
    • Choi KD, Yu J, Smuga-Otto K, et al. Hematopoietic and endothelial differentiation of human induced pluripotent stem cells. Stem Cells. 2009;27(3):559-567.
    • (2009) Stem Cells , vol.27 , Issue.3 , pp. 559-567
    • Choi, K.D.1    Yu, J.2    Smuga-Otto, K.3
  • 55
    • 84863116293 scopus 로고    scopus 로고
    • Generation of CD34+ cells from CCR5-disrupted human embryonic and induced pluripotent stem cells
    • Yao Y, Nashun B, Zhou T, et al. Generation of CD34+ cells from CCR5-disrupted human embryonic and induced pluripotent stem cells. Hum Gene Ther. 2012;23(2):238-242.
    • (2012) Hum Gene Ther , vol.23 , Issue.2 , pp. 238-242
    • Yao, Y.1    Nashun, B.2    Zhou, T.3
  • 56
    • 84903729497 scopus 로고    scopus 로고
    • Seamless modification of wild-type induced pluripotent stem cells to the natural CCR5Δ32 mutation confers resistance to HIV infection
    • Ye L, Wang J, Beyer AI, et al. Seamless modification of wild-type induced pluripotent stem cells to the natural CCR5Δ32 mutation confers resistance to HIV infection. Proc Natl Acad Sci USA. 2014;111(26):9591-9596.
    • (2014) Proc Natl Acad Sci USA , vol.111 , Issue.26 , pp. 9591-9596
    • Ye, L.1    Wang, J.2    Beyer, A.I.3
  • 57
    • 84903212620 scopus 로고    scopus 로고
    • TALENs facilitate targeted genome editing in human cells with high specificity and low cytotoxicity
    • Mussolino C, Alzubi J, Fine EJ, et al. TALENs facilitate targeted genome editing in human cells with high specificity and low cytotoxicity. Nucleic Acids Res. 2014;42(10):6762-6773.
    • (2014) Nucleic Acids Res , vol.42 , Issue.10 , pp. 6762-6773
    • Mussolino, C.1    Alzubi, J.2    Fine, E.J.3
  • 58
    • 84942921684 scopus 로고    scopus 로고
    • Efficient modification of CCR5 in primary human hematopoietic cells using a megaTAL nuclease and AAV donor template
    • Sather BD, Romano Ibarra GS, Sommer K, et al. Efficient modification of CCR5 in primary human hematopoietic cells using a megaTAL nuclease and AAV donor template. Sci Transl Med. 2015;7(307):307ra156.
    • (2015) Sci Transl Med , vol.7 , Issue.307
    • Sather, B.D.1    Romano Ibarra, G.S.2    Sommer, K.3
  • 59
    • 84922671463 scopus 로고    scopus 로고
    • Efficient ablation of genes in human hematopoietic stem and effector cells using CRISPR/Cas9
    • Mandal PK, Ferreira LM, Collins R, et al. Efficient ablation of genes in human hematopoietic stem and effector cells using CRISPR/Cas9. Cell Stem Cell. 2014;15(5):643-652.
    • (2014) Cell Stem Cell , vol.15 , Issue.5 , pp. 643-652
    • Mandal, P.K.1    Ferreira, L.M.2    Collins, R.3
  • 60
    • 84940203001 scopus 로고    scopus 로고
    • Inhibition of HIV-1 infection of primary CD4+ T-cells by gene editing of CCR5 using adenovirus-delivered CRISPR/Cas9
    • Li C, Guan X, Du T, et al. Inhibition of HIV-1 infection of primary CD4+ T-cells by gene editing of CCR5 using adenovirus-delivered CRISPR/Cas9. J Gen Virol. 2015;96(8):2381-2393.
    • (2015) J Gen Virol , vol.96 , Issue.8 , pp. 2381-2393
    • Li, C.1    Guan, X.2    Du, T.3
  • 61
    • 84937905397 scopus 로고    scopus 로고
    • Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells
    • Hendel A, Bak RO, Clark JT, et al. Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells. Nat Biotechnol. 2015;33(9):985-989.
    • (2015) Nat Biotechnol , vol.33 , Issue.9 , pp. 985-989
    • Hendel, A.1    Bak, R.O.2    Clark, J.T.3
  • 62
    • 0031575431 scopus 로고    scopus 로고
    • Change in coreceptor use correlates with disease progression in HIV-1-infected individuals
    • Connor RI, Sheridan KE, Ceradini D, Choe S, Landau NR. Change in coreceptor use correlates with disease progression in HIV-1-infected individuals. J Exp Med. 1997;185(4):621-628.
    • (1997) J Exp Med , vol.185 , Issue.4 , pp. 621-628
    • Connor, R.I.1    Sheridan, K.E.2    Ceradini, D.3    Choe, S.4    Landau, N.R.5
  • 63
    • 0033524834 scopus 로고    scopus 로고
    • Dependence of human stem cell engraftment and repopulation of NOD/SCID mice on CXCR4
    • Peled A, Petit I, Kollet O, et al. Dependence of human stem cell engraftment and repopulation of NOD/SCID mice on CXCR4. Science. 1999;283(5403):845-848.
    • (1999) Science , vol.283 , Issue.5403 , pp. 845-848
    • Peled, A.1    Petit, I.2    Kollet, O.3
  • 64
    • 79955785236 scopus 로고    scopus 로고
    • Engineering HIV-resistant human CD41 T cells with CXCR4-specific zinc-finger nucleases
    • Wilen CB, Wang J, Tilton JC, et al. Engineering HIV-resistant human CD41 T cells with CXCR4-specific zinc-finger nucleases. PLoS Pathog. 2011;7(4):e1002020.
    • (2011) PLoS Pathog , vol.7 , Issue.4
    • Wilen, C.B.1    Wang, J.2    Tilton, J.C.3
  • 65
    • 84862777608 scopus 로고    scopus 로고
    • Zinc-finger nuclease editing of human cxcr4 promotes HIV-1 CD4(+) T cell resistance and enrichment
    • Yuan J, Wang J, Crain K, et al. Zinc-finger nuclease editing of human cxcr4 promotes HIV-1 CD4(+) T cell resistance and enrichment. Mol Ther. 2012;20(4):849-859.
    • (2012) Mol Ther , vol.20 , Issue.4 , pp. 849-859
    • Yuan, J.1    Wang, J.2    Crain, K.3
  • 66
    • 84891772031 scopus 로고    scopus 로고
    • Simultaneous zinc-finger nuclease editing of the HIV coreceptors ccr5 and cxcr4 protects CD4+ T cells from HIV-1 infection
    • Didigu CA, Wilen CB, Wang J, et al. Simultaneous zinc-finger nuclease editing of the HIV coreceptors ccr5 and cxcr4 protects CD4+ T cells from HIV-1 infection. Blood. 2014;123(1):61-69.
    • (2014) Blood , vol.123 , Issue.1 , pp. 61-69
    • Didigu, C.A.1    Wilen, C.B.2    Wang, J.3
  • 67
    • 84878568502 scopus 로고    scopus 로고
    • Generation of an HIV resistant T-cell line by targeted "stacking" of restriction factors
    • Voit RA, McMahon MA, Sawyer SL, Porteus MH. Generation of an HIV resistant T-cell line by targeted "stacking" of restriction factors. Mol Ther. 2013;21(4):786-795.
    • (2013) Mol Ther , vol.21 , Issue.4 , pp. 786-795
    • Voit, R.A.1    McMahon, M.A.2    Sawyer, S.L.3    Porteus, M.H.4
  • 69
    • 1842732157 scopus 로고    scopus 로고
    • A single amino acid substitution in human APOBEC3G antiretroviral enzyme confers resistance to HIV-1 virion infectivity factor-induced depletion
    • Xu H, Svarovskaia ES, Barr R, et al. A single amino acid substitution in human APOBEC3G antiretroviral enzyme confers resistance to HIV-1 virion infectivity factor-induced depletion. Proc Natl Acad Sci USA. 2004;101(15):5652-5657.
    • (2004) Proc Natl Acad Sci USA , vol.101 , Issue.15 , pp. 5652-5657
    • Xu, H.1    Svarovskaia, E.S.2    Barr, R.3
  • 70
    • 11844278251 scopus 로고    scopus 로고
    • A single amino acid change in the SPRY domain of human Trim5alpha leads to HIV-1 restriction
    • Yap MW, Nisole S, Stoye JP. A single amino acid change in the SPRY domain of human Trim5alpha leads to HIV-1 restriction. Curr Biol. 2005;15(1):73-78.
    • (2005) Curr Biol , vol.15 , Issue.1 , pp. 73-78
    • Yap, M.W.1    Nisole, S.2    Stoye, J.P.3
  • 71
    • 67249100279 scopus 로고    scopus 로고
    • Mutation of a single residue renders human tetherin resistant to HIV-1 Vpu-mediated depletion
    • Gupta RK, Hué S, Schaller T, Verschoor E, Pillay D, Towers GJ. Mutation of a single residue renders human tetherin resistant to HIV-1 Vpu-mediated depletion. PLoS Pathog. 2009;5(5):e1000443.
    • (2009) PLoS Pathog , vol.5 , Issue.5
    • Gupta, R.K.1    Hué, S.2    Schaller, T.3    Verschoor, E.4    Pillay, D.5    Towers, G.J.6
  • 72
    • 78650381228 scopus 로고    scopus 로고
    • Genetic associations of variants in genes encoding HIV-dependency factors required for HIV-1 infection
    • Chinn LW, Tang M, Kessing BD, et al. Genetic associations of variants in genes encoding HIV-dependency factors required for HIV-1 infection. J Infect Dis. 2010;202(12):1836-1845.
    • (2010) J Infect Dis , vol.202 , Issue.12 , pp. 1836-1845
    • Chinn, L.W.1    Tang, M.2    Kessing, B.D.3
  • 74
    • 84949753192 scopus 로고    scopus 로고
    • Identification of novel HIV-1 dependency factors in primary CCR4(+)CCR6(+)Th17 cells via a genome-wide transcriptional approach
    • Cleret-Buhot A, Zhang Y, Planas D, et al. Identification of novel HIV-1 dependency factors in primary CCR4(+)CCR6(+)Th17 cells via a genome-wide transcriptional approach. Retrovirology. 2015;12(1):102.
    • (2015) Retrovirology , vol.12 , Issue.1 , pp. 102
    • Cleret-Buhot, A.1    Zhang, Y.2    Planas, D.3
  • 75
    • 34548134145 scopus 로고    scopus 로고
    • A whole-genome association study of major determinants for host control of HIV-1
    • Fellay J, Shianna KV, Ge D, et al. A whole-genome association study of major determinants for host control of HIV-1. Science. 2007; 317(5840):944-947.
    • (2007) Science , vol.317 , Issue.5840 , pp. 944-947
    • Fellay, J.1    Shianna, K.V.2    Ge, D.3
  • 76
    • 35948946526 scopus 로고    scopus 로고
    • Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery
    • Lombardo A, Genovese P, Beausejour CM, et al. Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery. Nat Biotechnol. 2007;25(11):1298-1306.
    • (2007) Nat Biotechnol , vol.25 , Issue.11 , pp. 1298-1306
    • Lombardo, A.1    Genovese, P.2    Beausejour, C.M.3
  • 77
    • 84898046908 scopus 로고    scopus 로고
    • Quantifying genome-editing outcomes at endogenous loci with SMRT sequencing
    • Hendel A, Kildebeck EJ, Fine EJ, et al. Quantifying genome-editing outcomes at endogenous loci with SMRT sequencing. Cell Reports. 2014;7(1):293-305.
    • (2014) Cell Reports , vol.7 , Issue.1 , pp. 293-305
    • Hendel, A.1    Kildebeck, E.J.2    Fine, E.J.3
  • 78
    • 18944373328 scopus 로고    scopus 로고
    • Highly efficient endogenous human gene correction using designed zinc-finger nucleases
    • Urnov FD, Miller JC, Lee YL, et al. Highly efficient endogenous human gene correction using designed zinc-finger nucleases. Nature. 2005;435(7042):646-651.
    • (2005) Nature , vol.435 , Issue.7042 , pp. 646-651
    • Urnov, F.D.1    Miller, J.C.2    Lee, Y.L.3
  • 79
    • 0347086143 scopus 로고    scopus 로고
    • In vivo selection of MGMT(P140K) lentivirus-transduced human NOD/SCID repopulating cells without pretransplant irradiation conditioning
    • Zielske SP, Reese JS, Lingas KT, Donze JR, Gerson SL. In vivo selection of MGMT(P140K) lentivirus-transduced human NOD/SCID repopulating cells without pretransplant irradiation conditioning. J Clin Invest. 2003;112(10):1561-1570.
    • (2003) J Clin Invest , vol.112 , Issue.10 , pp. 1561-1570
    • Zielske, S.P.1    Reese, J.S.2    Lingas, K.T.3    Donze, J.R.4    Gerson, S.L.5
  • 80
    • 1342290228 scopus 로고    scopus 로고
    • In vivo selection for human and murine hematopoietic cells transduced with a therapeutic MGMT lentiviral vector that inhibits HIV replication
    • Davis BM, Humeau L, Dropulic B. In vivo selection for human and murine hematopoietic cells transduced with a therapeutic MGMT lentiviral vector that inhibits HIV replication. Mol Ther. 2004;9(2):160-172.
    • (2004) Mol Ther , vol.9 , Issue.2 , pp. 160-172
    • Davis, B.M.1    Humeau, L.2    Dropulic, B.3
  • 81
    • 34347398120 scopus 로고    scopus 로고
    • HIV-1 proviral DNA excision using an evolved recombinase
    • Sarkar I, Hauber I, Hauber J, Buchholz F. HIV-1 proviral DNA excision using an evolved recombinase. Science. 2007;316(5833):1912-1915.
    • (2007) Science , vol.316 , Issue.5833 , pp. 1912-1915
    • Sarkar, I.1    Hauber, I.2    Hauber, J.3    Buchholz, F.4
  • 82
    • 84953243844 scopus 로고    scopus 로고
    • Universal Tre (uTre) recombinase specifically targets the majority of HIV-1 isolates
    • Karpinski J, Chemnitz J, Hauber I, et al. Universal Tre (uTre) recombinase specifically targets the majority of HIV-1 isolates. J Int AIDS Soc. 2014;17(4 Suppl 3):19706.
    • (2014) J Int AIDS Soc , vol.17 , Issue.4
    • Karpinski, J.1    Chemnitz, J.2    Hauber, I.3
  • 83
    • 84885008722 scopus 로고    scopus 로고
    • Zinc-finger-nucleases mediate specific and efficient excision of HIV-1 proviral DNA from infected and latently infected human T cells
    • Qu X, Wang P, Ding D, et al. Zinc-finger-nucleases mediate specific and efficient excision of HIV-1 proviral DNA from infected and latently infected human T cells. Nucleic Acids Res. 2013;41(16):7771-7782.
    • (2013) Nucleic Acids Res , vol.41 , Issue.16 , pp. 7771-7782
    • Qu, X.1    Wang, P.2    Ding, D.3
  • 84
    • 84883305437 scopus 로고    scopus 로고
    • Harnessing the CRISPR/Cas9 system to disrupt latent HIV-1 provirus
    • Ebina H, Misawa N, Kanemura Y, Koyanagi Y. Harnessing the CRISPR/Cas9 system to disrupt latent HIV-1 provirus. Sci Rep. 2013;3:2510.
    • (2013) Sci Rep , vol.3 , pp. 2510
    • Ebina, H.1    Misawa, N.2    Kanemura, Y.3    Koyanagi, Y.4
  • 85
    • 84905643812 scopus 로고    scopus 로고
    • RNA-directed gene editing specifically eradicates latent and prevents new HIV-1 infection
    • Hu W, Kaminski R, Yang F, et al. RNA-directed gene editing specifically eradicates latent and prevents new HIV-1 infection. Proc Natl Acad Sci USA. 2014;111(31):11461-11466.
    • (2014) Proc Natl Acad Sci USA , vol.111 , Issue.31 , pp. 11461-11466
    • Hu, W.1    Kaminski, R.2    Yang, F.3
  • 86
    • 84928739868 scopus 로고    scopus 로고
    • The CRISPR/Cas9 system inactivates latent HIV-1 proviral DNA
    • Zhu W, Lei R, Le Duff Y, et al. The CRISPR/Cas9 system inactivates latent HIV-1 proviral DNA. Retrovirology. 2015;12:22.
    • (2015) Retrovirology , vol.12 , pp. 22
    • Zhu, W.1    Lei, R.2    Le Duff, Y.3
  • 87
    • 84924943941 scopus 로고    scopus 로고
    • A high excision potential of TALENs for integrated DNA of HIV-based lentiviral vector
    • Ebina H, Kanemura Y, Misawa N, et al. A high excision potential of TALENs for integrated DNA of HIV-based lentiviral vector. PLoS One. 2015;10(3):e0120047.
    • (2015) PLoS One , vol.10 , Issue.3
    • Ebina, H.1    Kanemura, Y.2    Misawa, N.3
  • 88
    • 79951783813 scopus 로고    scopus 로고
    • Successful targeting and disruption of an integrated reporter lentivirus using the engineered homing endonuclease Y2 I-AniI
    • Aubert M, Ryu BY, Banks L, Rawlings DJ, Scharenberg AM, Jerome KR. Successful targeting and disruption of an integrated reporter lentivirus using the engineered homing endonuclease Y2 I-AniI. PLoS One. 2011;6(2):e16825.
    • (2011) PLoS One , vol.6 , Issue.2
    • Aubert, M.1    Ryu, B.Y.2    Banks, L.3    Rawlings, D.J.4    Scharenberg, A.M.5    Jerome, K.R.6
  • 89
    • 84939830659 scopus 로고    scopus 로고
    • Off-target-free gene delivery by affinity-purified receptor-targeted viral vectors
    • Münch RC, Muth A, Muik A, et al. Off-target-free gene delivery by affinity-purified receptor-targeted viral vectors. Nat Commun. 2015;6:6246.
    • (2015) Nat Commun , vol.6 , pp. 6246
    • Münch, R.C.1    Muth, A.2    Muik, A.3
  • 90
    • 84940105412 scopus 로고    scopus 로고
    • Exclusive transduction of human CD4+ T cells upon systemic delivery of CD4-targeted lentiviral vectors
    • Zhou Q, Uhlig KM, MuthA, et al. Exclusive transduction of human CD4+ T cells upon systemic delivery of CD4-targeted lentiviral vectors. J Immunol. 2015;195(5):2493-2501.
    • (2015) J Immunol , vol.195 , Issue.5 , pp. 2493-2501
    • Zhou, Q.1    Uhlig, K.M.2    Muth, A.3


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