메뉴 건너뛰기




Volumn 22, Issue 1, 2015, Pages

Generation of CCR5-defective CD34 cells from ZFN-driven stop codon-integrated mesenchymal stem cell clones

Author keywords

CCR5; Genome editing; HIV; Mesenchymal stem cells; Zinc finger nuclease

Indexed keywords

CD34 ANTIGEN; CD45 ANTIGEN; CHEMOKINE RECEPTOR CCR5; OCTAMER TRANSCRIPTION FACTOR 4; ZINC FINGER NUCLEASE; CCR5 PROTEIN, HUMAN; STOP CODON; ZINC FINGER PROTEIN;

EID: 84927720414     PISSN: 10217770     EISSN: 14230127     Source Type: Journal    
DOI: 10.1186/s12929-015-0130-6     Document Type: Article
Times cited : (16)

References (43)
  • 1
    • 0001633495 scopus 로고    scopus 로고
    • Genetic restriction of HIV-1 infection and progression to AIDS by a deletion allele of the CKR5 structural gene. Hemophilia growth and development study, multicenter AIDS cohort study, multicenter hemophilia cohort study, San Francisco city cohort, ALIVE study
    • Dean M, Carrington M, Winkler C, Huttley GA, Smith MW, Allikmets R, et al. Genetic restriction of HIV-1 infection and progression to AIDS by a deletion allele of the CKR5 structural gene. Hemophilia growth and development study, multicenter AIDS cohort study, multicenter hemophilia cohort study, San Francisco city cohort, ALIVE study. Science. 1996;273:1856-62.
    • (1996) Science , vol.273 , pp. 1856-1862
    • Dean, M.1    Carrington, M.2    Winkler, C.3    Huttley, G.A.4    Smith, M.W.5    Allikmets, R.6
  • 2
    • 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, Ceradini D, Martin SR, Horuk R, et al. Homozygous defect in HIV-1 coreceptor accounts for resistance of some multiply-exposed individuals to HIV-1 infection. Cell. 1996;86:367-77.
    • (1996) Cell , vol.86 , pp. 367-377
    • Liu, R.1    Paxton, W.A.2    Choe, S.3    Ceradini, D.4    Martin, S.R.5    Horuk, R.6
  • 4
    • 79953117041 scopus 로고    scopus 로고
    • Evidence for the cure of HIV infection by CCR5Δ32/Δ32 stem cell transplantation
    • Allers K, Hütter G, Hofmann J, Loddenkemper C, Rieger K, Thiel E, et al. Evidence for the cure of HIV infection by CCR5Δ32/Δ32 stem cell transplantation. Blood. 2011;117:2791-9.
    • (2011) Blood , vol.117 , pp. 2791-2799
    • Allers, K.1    Hütter, G.2    Hofmann, J.3    Loddenkemper, C.4    Rieger, K.5    Thiel, E.6
  • 5
    • 84878503062 scopus 로고    scopus 로고
    • Challenges in detecting HIV persistence during potentially curative interventions: A study of the Berlin patient
    • Yukl SA, Boritz E, Busch M, Bentsen C, Chun TW, Douek D, et al. Challenges in detecting HIV persistence during potentially curative interventions: a study of the Berlin patient. PLoS Pathog. 2013;9:e1003347.
    • (2013) PLoS Pathog , vol.9 , pp. e1003347
    • Yukl, S.A.1    Boritz, E.2    Busch, M.3    Bentsen, C.4    Chun, T.W.5    Douek, D.6
  • 6
    • 27844602105 scopus 로고    scopus 로고
    • The geographic spread of the CCR5 Delta32 HIV-resistance allele
    • Novembre J, Galvani AP, Slatkin M. The geographic spread of the CCR5 Delta32 HIV-resistance allele. PLoS Biol. 2005;3:e339.
    • (2005) PLoS Biol , vol.3 , pp. e339
    • Novembre, J.1    Galvani, A.P.2    Slatkin, M.3
  • 7
    • 33748094648 scopus 로고    scopus 로고
    • The Black Death and AIDS: CCR5-Delta32 in genetics and history
    • Cohn Jr SK, Weaver LT. The Black Death and AIDS: CCR5-Delta32 in genetics and history. QJM. 2006;99:497-503.
    • (2006) QJM , vol.99 , pp. 497-503
    • Cohn, S.K.1    Weaver, L.T.2
  • 8
    • 0037510038 scopus 로고    scopus 로고
    • Enhancing gene targeting with designed zinc finger nucleases
    • Bibikova M, Beumer K, Trautman JK, Carroll D. Enhancing gene targeting with designed zinc finger nucleases. Science. 2003;300:764.
    • (2003) Science , vol.300 , pp. 764
    • Bibikova, M.1    Beumer, K.2    Trautman, J.K.3    Carroll, D.4
  • 9
    • 55549129245 scopus 로고    scopus 로고
    • Progress and prospects: Zinc-finger nucleases as gene therapy agents
    • Carroll D. Progress and prospects: zinc-finger nucleases as gene therapy agents. Gene Ther. 2008;15:1463-8.
    • (2008) Gene Ther , vol.15 , pp. 1463-1468
    • Carroll, D.1
  • 10
    • 18944373328 scopus 로고    scopus 로고
    • Highly efficient endogenous human gene correction using designed zinc-finger nucleases
    • Urnov FD, Miller JC, Lee YL, Beausejour CM, Rock JM, Augustus S, et al. Highly efficient endogenous human gene correction using designed zinc-finger nucleases. Nature. 2005;435:646-51.
    • (2005) Nature , vol.435 , pp. 646-651
    • Urnov, F.D.1    Miller, J.C.2    Lee, Y.L.3    Beausejour, C.M.4    Rock, J.M.5    Augustus, S.6
  • 11
    • 44449120801 scopus 로고    scopus 로고
    • Targeted gene knockout in mammalian cells by using engineered zinc-finger nucleases
    • Santiago Y, Chan E, Liu PQ, Orlando S, Zhang L, Urnov FD, et al. Targeted gene knockout in mammalian cells by using engineered zinc-finger nucleases. Proc Natl Acad Sci. 2008;105:5809-14.
    • (2008) Proc Natl Acad Sci , vol.105 , pp. 5809-5814
    • Santiago, Y.1    Chan, E.2    Liu, P.Q.3    Orlando, S.4    Zhang, L.5    Urnov, F.D.6
  • 13
    • 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, Jouvenot Y, Kim KA, Liu O, et al. Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases. Nat Biotech. 2008;7:808-16.
    • (2008) Nat Biotech , vol.7 , pp. 808-816
    • Perez, E.E.1    Wang, J.2    Miller, J.C.3    Jouvenot, Y.4    Kim, K.A.5    Liu, O.6
  • 14
    • 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, Friedman G, Wang X, Taupin V, et al. Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo. Nat Biotechnol. 2010;28:839-47.
    • (2010) Nat Biotechnol , vol.28 , pp. 839-847
    • Holt, N.1    Wang, J.2    Kim, K.3    Friedman, G.4    Wang, X.5    Taupin, V.6
  • 15
    • 0038523969 scopus 로고    scopus 로고
    • Chimeric nucleases stimulate gene targeting in human cells
    • Porteus MH, 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
  • 16
    • 33847682924 scopus 로고    scopus 로고
    • Targeted gene addition into a specified location in the human genome using designed zinc finger nucleases
    • Moehle EA, Rock JM, Lee YL, Jouvenot Y, DeKelver RC, Gregory PD, et al. Targeted gene addition into a specified location in the human genome using designed zinc finger nucleases. Proc Natl Acad Sci U S A. 2007;104:3055-60.
    • (2007) Proc Natl Acad Sci U S A , vol.104 , pp. 3055-3060
    • Moehle, E.A.1    Rock, J.M.2    Lee, Y.L.3    Jouvenot, Y.4    DeKelver, R.C.5    Gregory, P.D.6
  • 17
    • 47349097567 scopus 로고    scopus 로고
    • Rapid "open-source" engineering of customized zinc-finger nucleases for highly efficient gene modification
    • Maeder ML, Thibodeau-Beganny S, Osiak A, Wright DA, Anthony RM, Eichtinger M, et al. Rapid "open-source" engineering of customized zinc-finger nucleases for highly efficient gene modification. Mol Cell. 2008;31:294-301.
    • (2008) Mol Cell , vol.31 , pp. 294-301
    • Maeder, M.L.1    Thibodeau-Beganny, S.2    Osiak, A.3    Wright, D.A.4    Anthony, R.M.5    Eichtinger, M.6
  • 18
    • 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 RC, Choi VM, Moehle EA, Paschon DE, Hockemeyer D, Meijsing SH, 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;8:1133-42.
    • (2010) Genome Res , vol.8 , 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
  • 20
    • 77950572073 scopus 로고    scopus 로고
    • Targeted gene addition to human mesenchymal stromal cells as a cell-based plasma-soluble protein delivery platform
    • Benabdallah BF, Allard E, Yao S, Friedman G, Friedman G, Gregory PD, et al. Targeted gene addition to human mesenchymal stromal cells as a cell-based plasma-soluble protein delivery platform. Cytotherapy. 2010;12:394-49.
    • (2010) Cytotherapy , vol.12 , pp. 394-449
    • Benabdallah, B.F.1    Allard, E.2    Yao, S.3    Friedman, G.4    Friedman, G.5    Gregory, P.D.6
  • 21
    • 79960833952 scopus 로고    scopus 로고
    • Generation of isogenic pluripotent stem cells differing exclusively at two early onset Parkinson point mutations
    • Soldner F, Laganière J, Cheng AW, Hockemeyer D, Gao Q, Alagappan R, et al. Generation of isogenic pluripotent stem cells differing exclusively at two early onset Parkinson point mutations. Cell. 2011;146:318-31.
    • (2011) Cell , vol.146 , pp. 318-331
    • Soldner, F.1    Laganière, J.2    Cheng, A.W.3    Hockemeyer, D.4    Gao, Q.5    Alagappan, R.6
  • 22
    • 0017119380 scopus 로고
    • Fibroblast precursors in normal and irradiated mouse hematopoietic organs
    • Friedenstein AJ, Gorskaja JF, Kulagina NN. Fibroblast precursors in normal and irradiated mouse hematopoietic organs. Exp Hematol. 1976;4:267-74.
    • (1976) Exp Hematol , vol.4 , pp. 267-274
    • Friedenstein, A.J.1    Gorskaja, J.F.2    Kulagina, N.N.3
  • 23
    • 0018947660 scopus 로고
    • Characterization of human bone marrow fibroblast colony-forming cells (CFU-F) and their progeny
    • Castro-Malaspina H, Gay RE, Resnick G, Kapoor N, Meyers P, Chiarieri D, et al. Characterization of human bone marrow fibroblast colony-forming cells (CFU-F) and their progeny. Blood. 1980;56:289-301.
    • (1980) Blood , vol.56 , pp. 289-301
    • Castro-Malaspina, H.1    Gay, R.E.2    Resnick, G.3    Kapoor, N.4    Meyers, P.5    Chiarieri, D.6
  • 24
    • 33747195353 scopus 로고    scopus 로고
    • Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors
    • Takahashi K, Yamanaka S. Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors. Cell. 2006;126:663-76.
    • (2006) Cell , vol.126 , pp. 663-676
    • Takahashi, K.1    Yamanaka, S.2
  • 25
    • 84885920900 scopus 로고    scopus 로고
    • Targeted transgene insertion into the AAVS1 locus driven by baculoviral vector-mediated zinc finger nuclease expression in human-induced pluripotent stem cells
    • Tay FC, Tan WK, Goh SL, Ramachandra CJ, Lau CH, Zhu H, et al. Targeted transgene insertion into the AAVS1 locus driven by baculoviral vector-mediated zinc finger nuclease expression in human-induced pluripotent stem cells. J Gene Med. 2013;15:384-95.
    • (2013) J Gene Med , vol.15 , pp. 384-395
    • Tay, F.C.1    Tan, W.K.2    Goh, S.L.3    Ramachandra, C.J.4    Lau, C.H.5    Zhu, H.6
  • 26
    • 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, Qin L, Qin L, Zhao S, et al. Generation of CD34+ cells from CCR5-disrupted human embryonic and induced pluripotent stem cells. Hum Gene Ther. 2012;23:23842.
    • (2012) Hum Gene Ther , vol.23 , pp. 23842
    • Yao, Y.1    Nashun, B.2    Zhou, T.3    Qin, L.4    Qin, L.5    Zhao, S.6
  • 27
    • 84895528015 scopus 로고    scopus 로고
    • Isolation of single-base genome-edited human iPS cells without antibiotic selection
    • Miyaoka Y, Chan AH, Judge LM, Yoo J, Huang M, Nguyen TD, et al. Isolation of single-base genome-edited human iPS cells without antibiotic selection. Nat Methods. 2014;11:291-3.
    • (2014) Nat Methods , vol.11 , pp. 291-293
    • Miyaoka, Y.1    Chan, A.H.2    Judge, L.M.3    Yoo, J.4    Huang, M.5    Nguyen, T.D.6
  • 28
    • 84873339687 scopus 로고    scopus 로고
    • Generation and genetic engineering of human induced pluripotent stem cells using designed zinc finger nucleases
    • Ramalingam S, London V, Kandavelou K, Cebotaru L, Guggino W, Civin C, et al. Generation and genetic engineering of human induced pluripotent stem cells using designed zinc finger nucleases. Stem Cells Dev. 2013;22:595-610.
    • (2013) Stem Cells Dev , vol.22 , pp. 595-610
    • Ramalingam, S.1    London, V.2    Kandavelou, K.3    Cebotaru, L.4    Guggino, W.5    Civin, C.6
  • 29
    • 71449125977 scopus 로고    scopus 로고
    • Forcing cells to change lineages
    • Graf T, Enver T. Forcing cells to change lineages. Nature. 2009;462:587-94.
    • (2009) Nature , vol.462 , pp. 587-594
    • Graf, T.1    Enver, T.2
  • 31
    • 4444263350 scopus 로고    scopus 로고
    • Isolation of a highly clonogenic and multipotential subfraction of adult stem cells from bone marrow stroma
    • Smith JR, Pochampally R, Perry A, Hsu SC, Prockop DJ. Isolation of a highly clonogenic and multipotential subfraction of adult stem cells from bone marrow stroma. Stem Cells. 2001;22:823-31.
    • (2001) Stem Cells , vol.22 , pp. 823-831
    • Smith, J.R.1    Pochampally, R.2    Perry, A.3    Hsu, S.C.4    Prockop, D.J.5
  • 32
    • 34948849730 scopus 로고    scopus 로고
    • Human platelet lysate allows expansion and clinical grade production of mesenchymal stromal cells from small samples of bone marrow aspirates or marrow filter washouts
    • Capelli C, Domenghini M, Borleri G, Bellavita P, Poma R, Carobbio A, et al. Human platelet lysate allows expansion and clinical grade production of mesenchymal stromal cells from small samples of bone marrow aspirates or marrow filter washouts. Bone Marrow Transplant. 2007;40:785-91.
    • (2007) Bone Marrow Transplant , vol.40 , pp. 785-791
    • Capelli, C.1    Domenghini, M.2    Borleri, G.3    Bellavita, P.4    Poma, R.5    Carobbio, A.6
  • 35
    • 67650045497 scopus 로고    scopus 로고
    • Targeted genome editing in human cells with zinc finger nucleases constructed via modular assembly
    • Kim HJ, Lee HJ, Kim H, Cho SW, Kim JS. Targeted genome editing in human cells with zinc finger nucleases constructed via modular assembly. Genome Res. 2009;19:1279-88.
    • (2009) Genome Res , vol.19 , pp. 1279-1288
    • Kim, H.J.1    Lee, H.J.2    Kim, H.3    Cho, S.W.4    Kim, J.S.5
  • 36
    • 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, Colleoni S, Lee YL, KimKA, et al. Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery. Nat Biotechnol. 2007;25:1298-306.
    • (2007) Nat Biotechnol , vol.25 , pp. 1298-1306
    • Lombardo, A.1    Genovese, P.2    Beausejour, C.M.3    Colleoni, S.4    Lee, Y.L.5    Kim, K.A.6
  • 37
    • 84874185862 scopus 로고    scopus 로고
    • Magnetic separation and antibiotics selection enable enrichment of cells with ZFN/TALEN-induced mutations
    • Kim H, Kim MS, Wee G, Lee CI, Kim H, Kim JS. Magnetic separation and antibiotics selection enable enrichment of cells with ZFN/TALEN-induced mutations. PLoS One. 2013;8:e56476.
    • (2013) PLoS One , vol.8 , pp. e56476
    • Kim, H.1    Kim, M.S.2    Wee, G.3    Lee, C.I.4    Kim, H.5    Kim, J.S.6
  • 38
    • 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, Frank I, Wang SQ, Lee G, et al. Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV. N Engl J Med. 2014;370:901-10.
    • (2014) N Engl J Med , vol.370 , pp. 901-910
    • Tebas, P.1    Stein, D.2    Tang, W.W.3    Frank, I.4    Wang, S.Q.5    Lee, G.6
  • 39
    • 84875420616 scopus 로고    scopus 로고
    • Efficient clinical scale gene modification via zinc finger nuclease-targeted disruption of the HIV co-receptor CCR5
    • Maier DA, Brennan AL, Jiang S, Binder-Scholl GK, Lee G, Plesa G, et al. Efficient clinical scale gene modification via zinc finger nuclease-targeted disruption of the HIV co-receptor CCR5. Hum Gene Ther. 2013;24:245-58.
    • (2013) Hum Gene Ther , vol.24 , pp. 245-258
    • Maier, D.A.1    Brennan, A.L.2    Jiang, S.3    Binder-Scholl, G.K.4    Lee, G.5    Plesa, G.6
  • 40
    • 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, Henley J, Rao A, Cao LF, et al. Genomic editing of the HIV-1 coreceptor CCR5 in adult hematopoietic stem and progenitor cells using zinc finger nucleases. Mol Ther. 2013;21:1259-69.
    • (2013) Mol Ther , vol.21 , pp. 1259-1269
    • Li, L.1    Krymskaya, L.2    Wang, J.3    Henley, J.4    Rao, A.5    Cao, L.F.6
  • 41
    • 0032079497 scopus 로고    scopus 로고
    • High CD34(+) cell counts decrease hematologic toxicity of autologous peripheral blood progenitor cell transplantation
    • Ketterer N, Salles G, Raba M, Espinouse D, Sonet A, Tremisi P, et al. High CD34(+) cell counts decrease hematologic toxicity of autologous peripheral blood progenitor cell transplantation. Blood. 1998;91:3148-55.
    • (1998) Blood , vol.91 , pp. 3148-3155
    • Ketterer, N.1    Salles, G.2    Raba, M.3    Espinouse, D.4    Sonet, A.5    Tremisi, P.6
  • 42
    • 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, Teque F, Cradick TJ, Qi Z, 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 U S A. 2014;111:9591-6.
    • (2014) Proc Natl Acad Sci U S A , vol.111 , pp. 9591-9596
    • Ye, L.1    Wang, J.2    Beyer, A.I.3    Teque, F.4    Cradick, T.J.5    Qi, Z.6
  • 43
    • 84904437964 scopus 로고    scopus 로고
    • Molecular evidence for OCT4 induced plasticity in adult human fibroblasts required for direct cell fate conversion to lineage specific progenitors
    • Mitchell R, Szabo E, Shapovalova Z, Aslostovar L, Makondo K, Bhatia M. Molecular evidence for OCT4 induced plasticity in adult human fibroblasts required for direct cell fate conversion to lineage specific progenitors. Stem Cells. 2014;32:2178-87.
    • (2014) Stem Cells , vol.32 , pp. 2178-2187
    • Mitchell, R.1    Szabo, E.2    Shapovalova, Z.3    Aslostovar, L.4    Makondo, K.5    Bhatia, M.6


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.