-
2
-
-
0037129435
-
Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy
-
DOI 10.1056/NEJMoa012616
-
Hacein-Bey-Abina S, Le Deist F, Carlier F, Bouneaud C, Hue C, De Villartay JP et al. Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. New Engl J Med 2002; 346: 1185-1193. (Pubitemid 34984579)
-
(2002)
New England Journal of Medicine
, vol.346
, Issue.16
, pp. 1185-1193
-
-
Hacein-Bey-Abina, S.1
Le Deist, F.2
Carlier, F.3
Bouneaud, C.4
Hue, C.5
De Villartay, J.-P.6
Thrasher, A.J.7
Wulffraat, N.8
Sorensen, R.9
Dupuis-Girod, S.10
Fischer, A.11
Graham Davies, E.12
Kuis, W.13
Leiva, L.14
Cavazzana-Calvo, M.15
-
3
-
-
0037189401
-
Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning
-
Aiuti A, Slavin S, Aker M, Ficara F, Deola S, Mortellaro A et al. Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science 2002; 296: 2410-2413.
-
(2002)
Science
, vol.296
, pp. 2410-2413
-
-
Aiuti, A.1
Slavin, S.2
Aker, M.3
Ficara, F.4
Deola, S.5
Mortellaro, A.6
-
4
-
-
59449098985
-
Gene therapy for immunodeficiency due to adenosine deaminase deficiency
-
Aiuti A, Cattaneo F, Galimberti S, Benninghoff U, Cassani B, Callegaro L et al. Gene therapy for immunodeficiency due to adenosine deaminase deficiency. N Engl J Med 2009; 360: 447-458.
-
(2009)
N Engl J Med
, vol.360
, pp. 447-458
-
-
Aiuti, A.1
Cattaneo, F.2
Galimberti, S.3
Benninghoff, U.4
Cassani, B.5
Callegaro, L.6
-
5
-
-
33645734405
-
Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1
-
Ott MG, Schmidt M, Schwarzwaelder K, Stein S, Siler U, Koehl U et al. Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nat Med 2006; 12: 401-409.
-
(2006)
Nat Med
, vol.12
, pp. 401-409
-
-
Ott, M.G.1
Schmidt, M.2
Schwarzwaelder, K.3
Stein, S.4
Siler, U.5
Koehl, U.6
-
6
-
-
0037448352
-
A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency
-
Hacein-Bey-Abina S, von Kalle C, Schmidt M, Le Deist F, Wulffraat N, McIntyre E et al. A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. New Engl J Med 2003; 348: 255-256.
-
(2003)
New Engl J Med
, vol.348
, pp. 255-256
-
-
Hacein-Bey-Abina, S.1
Von Kalle, C.2
Schmidt, M.3
Le Deist, F.4
Wulffraat, N.5
McIntyre, E.6
-
7
-
-
41149133413
-
Physiological promoters reduce the genotoxic risk of integrating gene vectors
-
Zychlinski D, Schambach A, Modlich U, Maetzig T, Meyer J, Grassman E et al. Physiological promoters reduce the genotoxic risk of integrating gene vectors. Mol Ther 2008; 16: 718-725.
-
(2008)
Mol Ther
, vol.16
, pp. 718-725
-
-
Zychlinski, D.1
Schambach, A.2
Modlich, U.3
Maetzig, T.4
Meyer, J.5
Grassman, E.6
-
8
-
-
33745108790
-
Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration
-
Montini E, Cesana D, Schmidt M, Sanvito F, Ponzoni M, Bartholomae C et al. Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration. Nat Biotechnol 2006; 24: 687-696.
-
(2006)
Nat Biotechnol
, vol.24
, pp. 687-696
-
-
Montini, E.1
Cesana, D.2
Schmidt, M.3
Sanvito, F.4
Ponzoni, M.5
Bartholomae, C.6
-
9
-
-
65249101260
-
The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy
-
Montini E, Cesana D, Schmidt M, Sanvito F, Bartholomae CC, Ranzani M et al. The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy. J Clin Invest 2009; 119: 964-975.
-
(2009)
J Clin Invest
, vol.119
, pp. 964-975
-
-
Montini, E.1
Cesana, D.2
Schmidt, M.3
Sanvito, F.4
Bartholomae, C.C.5
Ranzani, M.6
-
10
-
-
33750624015
-
Cell-culture assays reveal the importance of retroviral vector design for insertional genotoxicity
-
Modlich U, Bohne J, Schmidt M, von Kalle C, Knoss S, Schambach A et al. Cell-culture assays reveal the importance of retroviral vector design for insertional genotoxicity. Blood 2006; 108: 2545-2553.
-
(2006)
Blood
, vol.108
, pp. 2545-2553
-
-
Modlich, U.1
Bohne, J.2
Schmidt, M.3
Von Kalle, C.4
Knoss, S.5
Schambach, A.6
-
11
-
-
0031710033
-
A third-generation lentivirus vector with a conditional packaging system
-
Dull T, Zufferey R, Kelly M, Mandel RJ, Nguyen M, Trono D et al. A third-generation lentivirus vector with a conditional packaging system. J Virol 1998; 72: 8463-8471.
-
(1998)
J Virol
, vol.72
, pp. 8463-8471
-
-
Dull, T.1
Zufferey, R.2
Kelly, M.3
Mandel, R.J.4
Nguyen, M.5
Trono, D.6
-
12
-
-
0036881616
-
Vectors derived from simian immunodeficiency virus (SIV)
-
DOI 10.1016/S0300-9084(02)00036-6, PII S0300908402000366
-
Negre D, Cosset FL. Vectors derived from simian immunodeficiency virus (SIV). Biochimie 2002; 84: 1161-1171. (Pubitemid 36253742)
-
(2002)
Biochimie
, vol.84
, Issue.11
, pp. 1161-1171
-
-
Negre, D.1
Cosset, F.-L.2
-
13
-
-
0346132122
-
Self-inactivating retroviral vectors designed for transfer of whole genes into mammalian cells
-
Yu SF, von Ruden T, Kantoff PW, Garber C, Seiberg M, Ruther U et al. Self-inactivating retroviral vectors designed for transfer of whole genes into mammalian cells. Proc Natl Acad Sci USA 1986; 83: 3194-3198.
-
(1986)
Proc Natl Acad Sci USA
, vol.83
, pp. 3194-3198
-
-
Yu, S.F.1
Von Ruden, T.2
Kantoff, P.W.3
Garber, C.4
Seiberg, M.5
Ruther, U.6
-
14
-
-
0034322479
-
Context dependence of different modules for post-transcriptional enhancement of gene expression from retroviral vectors
-
Schambach A, Wodrich H, Hildinger M, Bohne J, Krausslich HG, Baum C. Context dependence of different modules for post-transcriptional enhancement of gene expression from retroviral vectors. Mol Ther 2000; 2: 435-445.
-
(2000)
Mol Ther
, vol.2
, pp. 435-445
-
-
Schambach, A.1
Wodrich, H.2
Hildinger, M.3
Bohne, J.4
Krausslich, H.G.5
Baum, C.6
-
15
-
-
30344464717
-
Equal potency of gammaretroviral and lentiviral SIN vectors for expression of O6-methylguanine-DNA methyltransferase in hematopoietic cells
-
Schambach A, Bohne J, Chandra S, Will E, Margison GP, Williams DA et al. Equal potency of gammaretroviral and lentiviral SIN vectors for expression of O6-methylguanine-DNA methyltransferase in hematopoietic cells. Mol Ther 2006; 13: 391-400.
-
(2006)
Mol Ther
, vol.13
, pp. 391-400
-
-
Schambach, A.1
Bohne, J.2
Chandra, S.3
Will, E.4
Margison, G.P.5
Williams, D.A.6
-
16
-
-
60749132598
-
Design and production of retro-and lentiviral vectors for gene expression in hematopoietic cells
-
Schambach A, Swaney WP, Loo JC. Design and production of retro-and lentiviral vectors for gene expression in hematopoietic cells. Methods Mol Biol 2009; 506: 191-205.
-
(2009)
Methods Mol Biol
, vol.506
, pp. 191-205
-
-
Schambach, A.1
Swaney, W.P.2
Loo, J.C.3
-
17
-
-
0033790710
-
The transgeneticist's toolbox: Novel methods for the targeted modification of eukaryotic genomes
-
Bode J, Schlake T, Iber M, Schubeler D, Seibler J, Snezhkov E et al. The transgeneticist's toolbox: novel methods for the targeted modification of eukaryotic genomes. Biol Chem 2000; 381: 801-813.
-
(2000)
Biol Chem
, vol.381
, pp. 801-813
-
-
Bode, J.1
Schlake, T.2
Iber, M.3
Schubeler, D.4
Seibler, J.5
Snezhkov, E.6
-
18
-
-
36148961543
-
Road to precision: Recombinase-based targeting technologies for genome engineering
-
Wirth D, Gama-Norton L, Riemer P, Sandhu U, Schucht R, Hauser H. Road to precision: recombinase-based targeting technologies for genome engineering. Curr Opin Biotechnol 2007; 18: 411-419.
-
(2007)
Curr Opin Biotechnol
, vol.18
, pp. 411-419
-
-
Wirth, D.1
Gama-Norton, L.2
Riemer, P.3
Sandhu, U.4
Schucht, R.5
Hauser, H.6
-
19
-
-
33745668475
-
A new generation of retroviral producer cells: Predictable and stable virus production by Flp-mediated site-specific integration of retroviral vectors
-
Schucht R, Coroadinha AS, Zanta-Boussif MA, Verhoeyen E, Carrondo MJ, Hauser H et al. A new generation of retroviral producer cells: predictable and stable virus production by Flp-mediated site-specific integration of retroviral vectors. Mol Ther 2006; 14: 285-292.
-
(2006)
Mol Ther
, vol.14
, pp. 285-292
-
-
Schucht, R.1
Coroadinha, A.S.2
Zanta-Boussif, M.A.3
Verhoeyen, E.4
Carrondo, M.J.5
Hauser, H.6
-
20
-
-
33745220778
-
The use of recombinase mediated cassette exchange in retroviral vector producer cell lines: Predictability and efficiency by transgene exchange
-
Coroadinha AS, Schucht R, Gama-Norton L, Wirth D, Hauser H, Carrondo MJ. The use of recombinase mediated cassette exchange in retroviral vector producer cell lines: predictability and efficiency by transgene exchange. J Biotechnol 2006; 124: 457-468.
-
(2006)
J Biotechnol
, vol.124
, pp. 457-468
-
-
Coroadinha, A.S.1
Schucht, R.2
Gama-Norton, L.3
Wirth, D.4
Hauser, H.5
Carrondo, M.J.6
-
21
-
-
0034925739
-
Evaluation of retroviral vector design in defined chromosomal loci by Flp-mediated cassette replacement
-
Verhoeyen E, Hauser H, Wirth D. Evaluation of retroviral vector design in defined chromosomal loci by Flp-mediated cassette replacement. Hum Gene Ther 2001; 12: 933-944.
-
(2001)
Hum Gene Ther
, vol.12
, pp. 933-944
-
-
Verhoeyen, E.1
Hauser, H.2
Wirth, D.3
-
22
-
-
0025834275
-
Construction and properties of retrovirus packaging cells based on gibbon ape leukemia virus
-
Miller AD, Garcia JV, von Suhr N, Lynch CM, Wilson C, Eiden MV. Construction and properties of retrovirus packaging cells based on gibbon ape leukemia virus. J Virol 1991; 65: 2220-2224.
-
(1991)
J Virol
, vol.65
, pp. 2220-2224
-
-
Miller, A.D.1
Garcia, J.V.2
Von Suhr, N.3
Lynch, C.M.4
Wilson, C.5
Eiden, M.V.6
-
23
-
-
19944421384
-
Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector
-
Gaspar HB, Parsley KL, Howe S, King D, Gilmour KC, Sinclair J et al. Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector. Lancet 2004; 364: 2181-2187.
-
(2004)
Lancet
, vol.364
, pp. 2181-2187
-
-
Gaspar, H.B.1
Parsley, K.L.2
Howe, S.3
King, D.4
Gilmour, K.C.5
Sinclair, J.6
-
24
-
-
0028109636
-
Use of mutated FLP recognition target (FRT) sites for the exchange of expression cassettes at defined chromosomal loci
-
Schlake T, Bode J. Use of mutated FLP recognition target (FRT) sites for the exchange of expression cassettes at defined chromosomal loci. Biochemistry 1994; 33: 12746-12751.
-
(1994)
Biochemistry
, vol.33
, pp. 12746-12751
-
-
Schlake, T.1
Bode, J.2
-
25
-
-
0029164582
-
Effects of retroviral vector design on expression of human adenosine deaminase in murine bone marrow transplant recipients engrafted with genetically modified cells
-
Riviere I, Brose K, Mulligan RC. Effects of retroviral vector design on expression of human adenosine deaminase in murine bone marrow transplant recipients engrafted with genetically modified cells. Proc Natl Acad Sci USA 1995; 92: 6733-6737.
-
(1995)
Proc Natl Acad Sci USA
, vol.92
, pp. 6733-6737
-
-
Riviere, I.1
Brose, K.2
Mulligan, R.C.3
-
26
-
-
33750023988
-
Overcoming promoter competition in packaging cells improves production of self-inactivating retroviral vectors
-
Schambach A, Mueller D, Galla M, Verstegen MM, Wagemaker G, Loew R et al. Overcoming promoter competition in packaging cells improves production of self-inactivating retroviral vectors. Gene Therapy 2006; 13: 1524-1533.
-
(2006)
Gene Therapy
, vol.13
, pp. 1524-1533
-
-
Schambach, A.1
Mueller, D.2
Galla, M.3
Verstegen, M.M.4
Wagemaker, G.5
Loew, R.6
-
27
-
-
0023276694
-
Effect of internal viral sequences on the utility of retroviral vectors
-
Armentano D, Yu SF, Kantoff PW, von Ruden T, Anderson WF, Gilboa E. Effect of internal viral sequences on the utility of retroviral vectors. J Virol 1987; 61: 1647-1650.
-
(1987)
J Virol
, vol.61
, pp. 1647-1650
-
-
Armentano, D.1
Yu, S.F.2
Kantoff, P.W.3
Von Ruden, T.4
Anderson, W.F.5
Gilboa, E.6
-
28
-
-
0023752108
-
Identification of a signal in a murine retrovirus that is sufficient for packaging of nonretroviral RNA into virions
-
Adam MA, Miller AD. Identification of a signal in a murine retrovirus that is sufficient for packaging of nonretroviral RNA into virions. J Virol 1988; 62: 3802-3806.
-
(1988)
J Virol
, vol.62
, pp. 3802-3806
-
-
Adam, M.A.1
Miller, A.D.2
-
29
-
-
34249298695
-
Improving transcriptional termination of self-inactivating gamma-retroviral and lentiviral vectors
-
Schambach A, Galla M, Maetzig T, Loew R, Baum C. Improving transcriptional termination of self-inactivating gamma-retroviral and lentiviral vectors. Mol Ther 2007; 15: 1167-1173.
-
(2007)
Mol Ther
, vol.15
, pp. 1167-1173
-
-
Schambach, A.1
Galla, M.2
Maetzig, T.3
Loew, R.4
Baum, C.5
-
31
-
-
58149469932
-
Transgene optimization significantly improves SIN vector titers, gp91(phox) expression and reconstitution of superoxide production in X-CGD cells
-
Moreno-Carranza B, Gentsch M, Stein S, Schambach A, Santilli G, Rudolf E et al. Transgene optimization significantly improves SIN vector titers, gp91(phox) expression and reconstitution of superoxide production in X-CGD cells. Gene Therapy 2009; 16: 111-118.
-
(2009)
Gene Therapy
, vol.16
, pp. 111-118
-
-
Moreno-Carranza, B.1
Gentsch, M.2
Stein, S.3
Schambach, A.4
Santilli, G.5
Rudolf, E.6
-
32
-
-
39849110286
-
Self-inactivating gammaretroviral vectors for gene therapy of X-linked severe combined immunodeficiency
-
Thornhill SI, Schambach A, Howe SJ, Ulaganathan M, Grassman E, Williams D et al. Self-inactivating gammaretroviral vectors for gene therapy of X-linked severe combined immunodeficiency. Mol Ther 2008; 16: 590-598.
-
(2008)
Mol Ther
, vol.16
, pp. 590-598
-
-
Thornhill, S.I.1
Schambach, A.2
Howe, S.J.3
Ulaganathan, M.4
Grassman, E.5
Williams, D.6
-
33
-
-
0028839609
-
High-titer packaging cells producing recombinant retroviruses resistant to human serum
-
Cosset FL, Takeuchi Y, Battini JL, Weiss RA, Collins MK. High-titer packaging cells producing recombinant retroviruses resistant to human serum. J Virol 1995; 69: 7430-7436.
-
(1995)
J Virol
, vol.69
, pp. 7430-7436
-
-
Cosset, F.L.1
Takeuchi, Y.2
Battini, J.L.3
Weiss, R.A.4
Collins, M.K.5
-
34
-
-
0030868549
-
Versatile retrovirus vector systems for regulated gene expression in vitro and in vivo
-
Lindemann D, Patriquin E, Feng S, Mulligan RC. Versatile retrovirus vector systems for regulated gene expression in vitro and in vivo. Mol Med 1997; 3: 466-476.
-
(1997)
Mol Med
, vol.3
, pp. 466-476
-
-
Lindemann, D.1
Patriquin, E.2
Feng, S.3
Mulligan, R.C.4
-
35
-
-
0029844184
-
Induction of antigen-specific tumor immunity by genetic and cellular vaccines against MAGE: Enhanced tumor protection by coexpression of granulocyte-macrophage colony-stimulating factor and B7-1
-
Bueler H, Mulligan RC. Induction of antigen-specific tumor immunity by genetic and cellular vaccines against MAGE: enhanced tumor protection by coexpression of granulocyte-macrophage colony-stimulating factor and B7-1. Mol Med 1996; 2: 545-555.
-
(1996)
Mol Med
, vol.2
, pp. 545-555
-
-
Bueler, H.1
Mulligan, R.C.2
-
36
-
-
0028122668
-
Expression of human glucocerebrosidase in murine macrophages: Identification of efficient retroviral vectors
-
Freas-Lutz DL, Correll PH, Dougherty SF, Xu L, Pluznik DH, Karlsson S. Expression of human glucocerebrosidase in murine macrophages: identification of efficient retroviral vectors. Exp Hematol 1994; 22: 857-865.
-
(1994)
Exp Hematol
, vol.22
, pp. 857-865
-
-
Freas-Lutz, D.L.1
Correll, P.H.2
Dougherty, S.F.3
Xu, L.4
Pluznik, D.H.5
Karlsson, S.6
-
37
-
-
0026720075
-
Tight control of gene expression in mammalian cells by tetracycline-responsive promoters
-
Gossen M, Bujard H. Tight control of gene expression in mammalian cells by tetracycline-responsive promoters. Proc Natl Acad Sci USA 1992; 89: 5547-5551.
-
(1992)
Proc Natl Acad Sci USA
, vol.89
, pp. 5547-5551
-
-
Gossen, M.1
Bujard, H.2
-
38
-
-
0028918657
-
A transient three-plasmid expression system for the production of high titer retroviral vectors
-
Soneoka Y, Cannon PM, Ramsdale EE, Griffiths JC, Romano G, Kingsman SM et al. A transient three-plasmid expression system for the production of high titer retroviral vectors. Nucleic Acids Res 1995; 23: 628-633.
-
(1995)
Nucleic Acids Res
, vol.23
, pp. 628-633
-
-
Soneoka, Y.1
Cannon, P.M.2
Ramsdale, E.E.3
Griffiths, J.C.4
Romano, G.5
Kingsman, S.M.6
-
39
-
-
0032976195
-
Foamy virus capsids require the cognate envelope protein for particle export
-
Pietschmann T, Heinkelein M, Heldmann M, Zentgraf H, Rethwilm A, Lindemann D. Foamy virus capsids require the cognate envelope protein for particle export. J Virol 1999; 73: 2613-2621.
-
(1999)
J Virol
, vol.73
, pp. 2613-2621
-
-
Pietschmann, T.1
Heinkelein, M.2
Heldmann, M.3
Zentgraf, H.4
Rethwilm, A.5
Lindemann, D.6
-
40
-
-
0023277545
-
Single-step method of RNA isolation by acid guanidinium thiocyanate-phenol-chloroform extraction
-
Chomczynski P, Sacchi N. Single-step method of RNA isolation by acid guanidinium thiocyanate-phenol-chloroform extraction. Anal Biochem 1987; 162: 156-159.
-
(1987)
Anal Biochem
, vol.162
, pp. 156-159
-
-
Chomczynski, P.1
Sacchi, N.2
-
41
-
-
0028248861
-
Sensitive, nonradioactive northern blots using alkaline transfer of total RNA and PCR-amplified biotinylated probes
-
1030
-
Loew R, Rausch T. Sensitive, nonradioactive northern blots using alkaline transfer of total RNA and PCR-amplified biotinylated probes. Biotechniques 1994; 17: 1026-1028, 1030.
-
(1994)
Biotechniques
, vol.17
, pp. 1026-1028
-
-
Loew, R.1
Rausch, T.2
-
42
-
-
76749093353
-
Retroviral vector performance upon integration in to defined chromosomal loci of modular packaging cell lines
-
Gama-Norton L, Herrmann S, Schucht R, Coroadinha A, Loew R, Alves P et al. Retroviral vector performance upon integration in to defined chromosomal loci of modular packaging cell lines. Hum Gene Ther (submitted).
-
Hum Gene Ther (Submitted)
-
-
Gama-Norton, L.1
Herrmann, S.2
Schucht, R.3
Coroadinha, A.4
Loew, R.5
Alves, P.6
|