-
1
-
-
84859790300
-
New tools in regenerative medicine: gene therapy
-
Munoz Ruiz M, Regueiro JR. New tools in regenerative medicine: gene therapy. Adv Exp Med Biol 2012; 741: 254-275.
-
(2012)
Adv Exp Med Biol
, vol.741
, pp. 254-275
-
-
Munoz Ruiz, M.1
Regueiro, J.R.2
-
2
-
-
0142084745
-
LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1
-
Hacein-Bey-Abina S, Kalle VC, Schmidt M, et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 2003; 302: 415-419.
-
(2003)
Science
, vol.302
, pp. 415-419
-
-
Hacein-Bey-Abina, S.1
Kalle, V.C.2
Schmidt, M.3
-
3
-
-
3042732990
-
Retrovirus silencing, variegation, extinction, and memory are controlled by a dynamic interplay of multiple epigenetic modifications
-
Yao S, Sukonnik T, Kean T, et al. Retrovirus silencing, variegation, extinction, and memory are controlled by a dynamic interplay of multiple epigenetic modifications. Mol Ther 2004; 10: 27-36.
-
(2004)
Mol Ther
, vol.10
, pp. 27-36
-
-
Yao, S.1
Sukonnik, T.2
Kean, T.3
-
4
-
-
27944496521
-
Retroviral insertional mutagenesis: past, present and future
-
Uren AG, Kool J, Berns A, et al. Retroviral insertional mutagenesis: past, present and future. Oncogene 2005; 24: 7656-7672.
-
(2005)
Oncogene
, vol.24
, pp. 7656-7672
-
-
Uren, A.G.1
Kool, J.2
Berns, A.3
-
6
-
-
80051535219
-
Genome engineering with zinc-finger nucleases
-
Carroll D. Genome engineering with zinc-finger nucleases. Genetics 2011; 188: 773-782.
-
(2011)
Genetics
, vol.188
, pp. 773-782
-
-
Carroll, D.1
-
7
-
-
77955867185
-
Genome editing with engineered zinc finger nucleases
-
Urnov FD, Rebar EJ, Holmes MC, et al. Genome editing with engineered zinc finger nucleases. Nat Rev Genet 2010; 11: 636-646.
-
(2010)
Nat Rev Genet
, vol.11
, pp. 636-646
-
-
Urnov, F.D.1
Rebar, E.J.2
Holmes, M.C.3
-
8
-
-
40949126488
-
Robust, persistent transgene expression in human embryonic stem cells is achieved with AAVS1-targeted integration
-
Smith JR, Maguire S, Davis LA, et al. Robust, persistent transgene expression in human embryonic stem cells is achieved with AAVS1-targeted integration. Stem Cells 2008; 26: 496-504.
-
(2008)
Stem Cells
, vol.26
, pp. 496-504
-
-
Smith, J.R.1
Maguire, S.2
Davis, L.A.3
-
9
-
-
70249114821
-
Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases
-
Hockemeyer D, Soldner F, Beard C, et al. Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases. Nat biotechnol 2009; 27: 851-857.
-
(2009)
Nat biotechnol
, vol.27
, pp. 851-857
-
-
Hockemeyer, D.1
Soldner, F.2
Beard, C.3
-
10
-
-
79955584622
-
Gene editing of human embryonic stem cells via an engineered baculoviral vector carrying zinc-finger nucleases
-
Lei Y, Lee CL, Joo K, et al. Gene editing of human embryonic stem cells via an engineered baculoviral vector carrying zinc-finger nucleases. Mol Ther 2011; 19: 942-950.
-
(2011)
Mol Ther
, vol.19
, pp. 942-950
-
-
Lei, Y.1
Lee, C.L.2
Joo, K.3
-
11
-
-
80052468612
-
Efficient recombinase-mediated cassette exchange at the AAVS1 locus in human embryonic stem cells using baculoviral vectors
-
Ramachandra CJ, Shahbazi M, Kwang TW, et al. Efficient recombinase-mediated cassette exchange at the AAVS1 locus in human embryonic stem cells using baculoviral vectors. Nucleic acids Res 2011; 39: e107.
-
(2011)
Nucleic acids Res
, vol.39
-
-
Ramachandra, C.J.1
Shahbazi, M.2
Kwang, T.W.3
-
13
-
-
0026070440
-
Targeted integration of adeno-assoicated virus (AAV) into human chromosome 19
-
Samulski RJ, Zhu X, Xiao X, et al. Targeted integration of adeno-assoicated virus (AAV) into human chromosome 19. EMBO J 1991; 10: 3941-3950.
-
(1991)
EMBO J
, vol.10
, pp. 3941-3950
-
-
Samulski, R.J.1
Zhu, X.2
Xiao, X.3
-
14
-
-
77955157281
-
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, 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; 20: 1133-1142.
-
(2010)
Genome Res
, vol.20
, pp. 1133-1142
-
-
DeKelver, R.C.1
Choi, V.M.2
Moehle, E.A.3
-
15
-
-
78651337233
-
Genomic safe harbors permit high beta-globin transgene expression in thalassemia induced pluripotent stem cells
-
Papapetrou EP, Lee G, Malani N, et al. Genomic safe harbors permit high beta-globin transgene expression in thalassemia induced pluripotent stem cells. Nat Biotechnol 2011; 29: 73-78.
-
(2011)
Nat Biotechnol
, vol.29
, pp. 73-78
-
-
Papapetrou, E.P.1
Lee, G.2
Malani, N.3
-
16
-
-
0042208122
-
Identification of an insulator in AAVS1, a preferred region for integration of adeno-associated virus DNA
-
Ogata T, Kozuka T, Kanda T. Identification of an insulator in AAVS1, a preferred region for integration of adeno-associated virus DNA. J Virol 2003; 77: 9000-9007.
-
(2003)
J Virol
, vol.77
, pp. 9000-9007
-
-
Ogata, T.1
Kozuka, T.2
Kanda, T.3
-
17
-
-
66149108014
-
Site-specific integration of adeno-associated virus involves partial duplication of the target locus
-
Henckaerts E, Dutheil N, Zeltner N, et al. Site-specific integration of adeno-associated virus involves partial duplication of the target locus. Proc Natl Acad Sci U S A 2009; 106: 7571-7576.
-
(2009)
Proc Natl Acad Sci U S A
, vol.106
, pp. 7571-7576
-
-
Henckaerts, E.1
Dutheil, N.2
Zeltner, N.3
-
18
-
-
80053350622
-
Site-specific integration and tailoring of cassette design for sustainable gene transfer
-
Lombardo A, Cesana D, Genovese P, et al. Site-specific integration and tailoring of cassette design for sustainable gene transfer. Nat Methods 2011; 8: 861-869.
-
(2011)
Nat Methods
, vol.8
, pp. 861-869
-
-
Lombardo, A.1
Cesana, D.2
Genovese, P.3
-
19
-
-
84873570368
-
Chromatin structure of two genomic sites for targeted transgene integration in induced pluripotent stem cells and haematopoietic stem cells
-
Van Rensburg R, Bever I, Yao XY, et al. Chromatin structure of two genomic sites for targeted transgene integration in induced pluripotent stem cells and haematopoietic stem cells. Gene Ther 2013; 20: 201-214.
-
(2013)
Gene Ther
, vol.20
, pp. 201-214
-
-
Van Rensburg, R.1
Bever, I.2
Yao, X.Y.3
-
20
-
-
34447319080
-
An improved zinc-finger nuclease architecture for highly specific genome editing
-
Miller JC, Holmes MC, Wang J, et al. An improved zinc-finger nuclease architecture for highly specific genome editing. Nat biotechnol 2007; 25: 778-785.
-
(2007)
Nat biotechnol
, vol.25
, pp. 778-785
-
-
Miller, J.C.1
Holmes, M.C.2
Wang, J.3
-
21
-
-
84860519007
-
Tumour tropism of intravenously injected human-induced pluripotent stem cell-derived neural stem cells and their gene therapy application in a metastatic breast cancer model
-
Yang J, Lam DH, Goh SS, et al. Tumour tropism of intravenously injected human-induced pluripotent stem cell-derived neural stem cells and their gene therapy application in a metastatic breast cancer model. Stem Cells 2012; 30: 1021-1029.
-
(2012)
Stem Cells
, vol.30
, pp. 1021-1029
-
-
Yang, J.1
Lam, D.H.2
Goh, S.S.3
-
22
-
-
84856952844
-
Targeted suicide gene therapy for glioma using human embryonic stem cell-derived neural stem cells genetically modified by baculoviral vectors
-
Zhao Y, Lam DH, Yang J, et al. Targeted suicide gene therapy for glioma using human embryonic stem cell-derived neural stem cells genetically modified by baculoviral vectors. Gene Ther 2012; 19: 189-200.
-
(2012)
Gene Ther
, vol.19
, pp. 189-200
-
-
Zhao, Y.1
Lam, D.H.2
Yang, J.3
-
23
-
-
36248966518
-
Induction of pluripotent stem cells from adult human fibroblasts by defined factors
-
Takahashi K, Tanabe K, Ohnuki M, et al. Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell 2007; 131: 861-872.
-
(2007)
Cell
, vol.131
, pp. 861-872
-
-
Takahashi, K.1
Tanabe, K.2
Ohnuki, M.3
-
24
-
-
34147097547
-
Baculoviral vector-mediated transient and stable transgene expression in human embryonic stem cells
-
Zeng J, Du J, Zhao Y, et al. Baculoviral vector-mediated transient and stable transgene expression in human embryonic stem cells. Stem Cells 2007; 25: 1055-1061.
-
(2007)
Stem Cells
, vol.25
, pp. 1055-1061
-
-
Zeng, J.1
Du, J.2
Zhao, Y.3
-
25
-
-
72249110432
-
The combined use of viral transcriptional and post-transcriptional regulatory elements to improve baculovirus-mediated transient gene expression in human embryonic stem cells
-
Du J, Zeng J, Zhao Y, et al. The combined use of viral transcriptional and post-transcriptional regulatory elements to improve baculovirus-mediated transient gene expression in human embryonic stem cells. J Biosci Bioeng 2010; 109: 1-8.
-
(2010)
J Biosci Bioeng
, vol.109
, pp. 1-8
-
-
Du, J.1
Zeng, J.2
Zhao, Y.3
-
26
-
-
80055102865
-
A graphene-based platform for induced pluripotent stem cells culture and differentiation
-
Chen GY, Pang DW, Hwang SM, et al. A graphene-based platform for induced pluripotent stem cells culture and differentiation. Biomaterials 2012; 33: 418-27.
-
(2012)
Biomaterials
, vol.33
, pp. 418-427
-
-
Chen, G.Y.1
Pang, D.W.2
Hwang, S.M.3
-
27
-
-
84864381319
-
Defective antiviral responses of induced pluripotent stem cells to baculoviral vector transduction
-
Chen GY, Hwang SM, Su HJ, et al. Defective antiviral responses of induced pluripotent stem cells to baculoviral vector transduction. J Virol 2012; 86: 8041-8049.
-
(2012)
J Virol
, vol.86
, pp. 8041-8049
-
-
Chen, G.Y.1
Hwang, S.M.2
Su, H.J.3
-
28
-
-
0034778062
-
Towards the use of baculovirus as a gene therapy vector
-
Pieroni L, La Monica N. Towards the use of baculovirus as a gene therapy vector. Curr Opin Mol Ther 2001; 3: 464-467.
-
(2001)
Curr Opin Mol Ther
, vol.3
, pp. 464-467
-
-
Pieroni, L.1
La Monica, N.2
-
29
-
-
0036532579
-
Recombinant baculoviruses as mammalian cell gene-delivery vectors
-
Kost TA, Condreay JP. Recombinant baculoviruses as mammalian cell gene-delivery vectors. Trends Biotechnol 2002; 20: 173-180.
-
(2002)
Trends Biotechnol
, vol.20
, pp. 173-180
-
-
Kost, T.A.1
Condreay, J.P.2
-
30
-
-
27744530147
-
Zinc finger nucleases: custom-designed molecular scissors for genome engineering of plant and mammalian cells
-
Durai S, Mani M, Kandavelou K, et al. Zinc finger nucleases: custom-designed molecular scissors for genome engineering of plant and mammalian cells. Nucleic Acids Res 2005; 33: 5978-5990.
-
(2005)
Nucleic Acids Res
, vol.33
, pp. 5978-5990
-
-
Durai, S.1
Mani, M.2
Kandavelou, K.3
-
31
-
-
34447323568
-
Structure-based redesign of the dimerization interface reduces the toxicity of zinc-finger nucleases
-
Szczepek M, Brondani V, Buchel J, et al. Structure-based redesign of the dimerization interface reduces the toxicity of zinc-finger nucleases. Nat Biotechnol 2007; 25: 786-793.
-
(2007)
Nat Biotechnol
, vol.25
, pp. 786-793
-
-
Szczepek, M.1
Brondani, V.2
Buchel, J.3
-
32
-
-
84871519181
-
TALENs: a widely applicable technology for targeted genome editing
-
Joung JK, Sander JD. TALENs: a widely applicable technology for targeted genome editing. Nat Rev Mol Cell Biol 2013; 14: 49-55.
-
(2013)
Nat Rev Mol Cell Biol
, vol.14
, pp. 49-55
-
-
Joung, J.K.1
Sander, J.D.2
-
33
-
-
84857032466
-
Structural basis for sequence-specific recognition of DNA by TAL effectors
-
Deng D, Yan C, Pan X, et al. Structural basis for sequence-specific recognition of DNA by TAL effectors. Science 2012; 335: 720-723.
-
(2012)
Science
, vol.335
, pp. 720-723
-
-
Deng, D.1
Yan, C.2
Pan, X.3
-
34
-
-
84857029597
-
The crystal structure of TAL effector PthXo1 bound to its DNA target
-
Mak AN, Bradley P, Cernadas RA, et al. The crystal structure of TAL effector PthXo1 bound to its DNA target. Science 2012; 335: 716-719.
-
(2012)
Science
, vol.335
, pp. 716-719
-
-
Mak, A.N.1
Bradley, P.2
Cernadas, R.A.3
-
35
-
-
72149110399
-
Breaking the code of DNA binding specificity of TAL-Type III effectors
-
Boch J, Scholze H, Schornack S, et al. Breaking the code of DNA binding specificity of TAL-Type III effectors. Science 2009; 326: 1509-1512.
-
(2009)
Science
, vol.326
, pp. 1509-1512
-
-
Boch, J.1
Scholze, H.2
Schornack, S.3
-
36
-
-
79960836456
-
Genetic engineering of human pluripotent cells using TALE nucleases
-
Hockemeyer D, Wang H, Kiani S, et al. Genetic engineering of human pluripotent cells using TALE nucleases. Nat Biotechnol 2011; 29: 731-734.
-
(2011)
Nat Biotechnol
, vol.29
, pp. 731-734
-
-
Hockemeyer, D.1
Wang, H.2
Kiani, S.3
-
37
-
-
84876389220
-
Differential integrity of TALE nuclease genes following adenoviral and lentiviral vector gene transfer into human cells
-
Holkers M, Maggio I, Liu J, et al. Differential integrity of TALE nuclease genes following adenoviral and lentiviral vector gene transfer into human cells. Nucleic Acids Res 2013; 41: e63.
-
(2013)
Nucleic Acids Res
, vol.41
-
-
Holkers, M.1
Maggio, I.2
Liu, J.3
-
38
-
-
84885939240
-
Baculoviral transduction faciliates TALEN-mediated targeted transgene integration and Cre/LoxP cassette exchange in human induced pluripotent stem cells
-
doi: 10.1093/nar/gkt721 [Epub ahead of print].
-
Zhu H, Lau CH, Goh SL, et al. Baculoviral transduction faciliates TALEN-mediated targeted transgene integration and Cre/LoxP cassette exchange in human induced pluripotent stem cells. Nucleic Acids Res 2013; doi: 10.1093/nar/gkt721 [Epub ahead of print].
-
(2013)
Nucleic Acids Res
-
-
Zhu, H.1
Lau, C.H.2
Goh, S.L.3
|