-
1
-
-
0027228422
-
Vesicular stomatitis virus G glycoprotein pseudotyped retroviral vectors: concentration to very high titer and efficient gene transfer into mammalian and non-mammalian cells
-
1 Burns, J.C., Friedmann, T., Driever, W., Burrascano, M., Yee, J.K., Vesicular stomatitis virus G glycoprotein pseudotyped retroviral vectors: concentration to very high titer and efficient gene transfer into mammalian and non-mammalian cells. Proc Natl Acad Sci USA 90 (1993), 8033–8037.
-
(1993)
Proc Natl Acad Sci USA
, vol.90
, pp. 8033-8037
-
-
Burns, J.C.1
Friedmann, T.2
Driever, W.3
Burrascano, M.4
Yee, J.K.5
-
2
-
-
33745328918
-
+ cells in non-human primates using GALV-pseudotyped gammaretroviral vectors
-
+ cells in non-human primates using GALV-pseudotyped gammaretroviral vectors. Mol Ther 14 (2006), 212–217.
-
(2006)
Mol Ther
, vol.14
, pp. 212-217
-
-
Beard, B.C.1
Mezquita, P.2
Morris, J.C.3
Kiem, H.P.4
-
3
-
-
0036682935
-
Lentiviral vectors pseudotyped with a modified RD114 envelope glycoprotein show increased stability in sera and augmented transduction of primary lymphocytes and CD34+ cells derived from human and nonhuman primates
-
3 Sandrin, V., Boson, B., Salmon, P., Gay, W., Nègre, D., Le Grand, R., Trono, D., Cosset, F.L., Lentiviral vectors pseudotyped with a modified RD114 envelope glycoprotein show increased stability in sera and augmented transduction of primary lymphocytes and CD34+ cells derived from human and nonhuman primates. Blood 100 (2002), 823–832.
-
(2002)
Blood
, vol.100
, pp. 823-832
-
-
Sandrin, V.1
Boson, B.2
Salmon, P.3
Gay, W.4
Nègre, D.5
Le Grand, R.6
Trono, D.7
Cosset, F.L.8
-
4
-
-
77950521575
-
Cocal-pseudotyped lentiviral vectors resist inactivation by human serum and efficiently transduce primate hematopoietic repopulating cells
-
4 Trobridge, G.D., Wu, R.A., Hansen, M., Ironside, C., Watts, K.L., Olsen, P., Beard, B.C., Kiem, H.P., Cocal-pseudotyped lentiviral vectors resist inactivation by human serum and efficiently transduce primate hematopoietic repopulating cells. Mol Ther 18 (2010), 725–733.
-
(2010)
Mol Ther
, vol.18
, pp. 725-733
-
-
Trobridge, G.D.1
Wu, R.A.2
Hansen, M.3
Ironside, C.4
Watts, K.L.5
Olsen, P.6
Beard, B.C.7
Kiem, H.P.8
-
5
-
-
84970028787
-
Development of third-generation cocal envelope producer cell lines for robust lentiviral gene transfer into hematopoietic stem cells and T-cells
-
[Epub ahead of print] The authors developed a third-generation cell line that produces high titer cocal pseudotyped vectors. The novel cocal pseudotyped vector provides broad tropism, resistance to human sera and low toxicity. These cocal pseudotyped producer cell lines should allow the production of large-scale clinical grade vector preparations, facilitating gene therapy trials.
-
5•• Humbert, O., Gisch, D.W., Wohlfahrt, M.E., Adams, A.B., Greenberg, P.D., Schmitt, T.M., Trobridge, G.D., Kiem, H.P., Development of third-generation cocal envelope producer cell lines for robust lentiviral gene transfer into hematopoietic stem cells and T-cells. Mol Ther, 2016 [Epub ahead of print] The authors developed a third-generation cell line that produces high titer cocal pseudotyped vectors. The novel cocal pseudotyped vector provides broad tropism, resistance to human sera and low toxicity. These cocal pseudotyped producer cell lines should allow the production of large-scale clinical grade vector preparations, facilitating gene therapy trials.
-
(2016)
Mol Ther
-
-
Humbert, O.1
Gisch, D.W.2
Wohlfahrt, M.E.3
Adams, A.B.4
Greenberg, P.D.5
Schmitt, T.M.6
Trobridge, G.D.7
Kiem, H.P.8
-
6
-
-
2342478423
-
Efficient lentiviral gene transfer to canine repopulating cells using an overnight transduction protocol
-
6 Horn, P.A., Keyser, K.A., Peterson, L.J., Neff, T., Thomasson, B.M., Thompson, J., Kiem, H.P., Efficient lentiviral gene transfer to canine repopulating cells using an overnight transduction protocol. Blood 103 (2004), 3710–3716.
-
(2004)
Blood
, vol.103
, pp. 3710-3716
-
-
Horn, P.A.1
Keyser, K.A.2
Peterson, L.J.3
Neff, T.4
Thomasson, B.M.5
Thompson, J.6
Kiem, H.P.7
-
7
-
-
47049090397
-
Efficient transduction of pigtailed macaque hematopoietic repopulating cells with HIV-based lentiviral vectors
-
7 Trobridge, G.D., Beard, B.C., Gooch, C., Wohlfahrt, M., Olsen, P., Fletcher, J., Malik, P., Kiem, H.P., Efficient transduction of pigtailed macaque hematopoietic repopulating cells with HIV-based lentiviral vectors. Blood 111 (2008), 5537–5543.
-
(2008)
Blood
, vol.111
, pp. 5537-5543
-
-
Trobridge, G.D.1
Beard, B.C.2
Gooch, C.3
Wohlfahrt, M.4
Olsen, P.5
Fletcher, J.6
Malik, P.7
Kiem, H.P.8
-
8
-
-
0029862497
-
A stable human-derived packaging cell line for production of high titer retrovirus/vesicular stomatitis virus G pseudotypes
-
8 Ory, D.S., Neugeboren, B.A., Mulligan, R.C., A stable human-derived packaging cell line for production of high titer retrovirus/vesicular stomatitis virus G pseudotypes. Proc Natl Acad Sci USA 93 (1996), 11400–11406.
-
(1996)
Proc Natl Acad Sci USA
, vol.93
, pp. 11400-11406
-
-
Ory, D.S.1
Neugeboren, B.A.2
Mulligan, R.C.3
-
9
-
-
0034264613
-
VSV-G pseudotyped lentiviral vector particles produced in human cells are inactivated by human serum
-
9 DePolo, N.J., Reed, J.D., Sheridan, P.L., Townsend, K., Sauter, S.L., Jolly, D.J., Dubensky, T.W. Jr., VSV-G pseudotyped lentiviral vector particles produced in human cells are inactivated by human serum. Mol Ther 2 (2000), 218–222.
-
(2000)
Mol Ther
, vol.2
, pp. 218-222
-
-
DePolo, N.J.1
Reed, J.D.2
Sheridan, P.L.3
Townsend, K.4
Sauter, S.L.5
Jolly, D.J.6
Dubensky, T.W.7
-
10
-
-
0027158091
-
Integration of murine leukemia virus DNA depends on mitosis
-
10 Roe, T., Reynolds, T.C., Yu, G., Brown, P.O., Integration of murine leukemia virus DNA depends on mitosis. EMBO J 12 (1993), 2099–2108.
-
(1993)
EMBO J
, vol.12
, pp. 2099-2108
-
-
Roe, T.1
Reynolds, T.C.2
Yu, G.3
Brown, P.O.4
-
11
-
-
0026654682
-
Human immunodeficiency virus infection of cells arrested in the cell cycle
-
11 Lewis, P., Hensel, M., Emerman, M., Human immunodeficiency virus infection of cells arrested in the cell cycle. EMBO J 11 (1992), 3053–3058.
-
(1992)
EMBO J
, vol.11
, pp. 3053-3058
-
-
Lewis, P.1
Hensel, M.2
Emerman, M.3
-
12
-
-
0031594498
-
Progression to the G1b phase of the cell cycle is required for completion of human immunodeficiency virus type 1 reverse transcription in T cells
-
12 Korin, Y.D., Zack, J.A., Progression to the G1b phase of the cell cycle is required for completion of human immunodeficiency virus type 1 reverse transcription in T cells. J Virol 72 (1998), 3161–3168.
-
(1998)
J Virol
, vol.72
, pp. 3161-3168
-
-
Korin, Y.D.1
Zack, J.A.2
-
13
-
-
1242296984
-
Cell cycle requirements for transduction by foamy virus vectors compared to those of oncovirus and lentivirus vectors
-
13 Trobridge, G., Russell, D.W., Cell cycle requirements for transduction by foamy virus vectors compared to those of oncovirus and lentivirus vectors. J Virol 78 (2004), 2327–2335.
-
(2004)
J Virol
, vol.78
, pp. 2327-2335
-
-
Trobridge, G.1
Russell, D.W.2
-
14
-
-
0032531012
-
Improved gene transfer into baboon marrow repopulating cells using recombinant human fibronectin fragment CH-296 in combination with interleukin-6, stem cell factor, FLT-3 ligand, and megakaryocyte growth and development factor
-
14 Kiem, H.P., Andrews, R.G., Morris, J., Peterson, L., Heyward, S., Allen, J.M., Rasko, J.E., Potter, J., Miller, A.D., Improved gene transfer into baboon marrow repopulating cells using recombinant human fibronectin fragment CH-296 in combination with interleukin-6, stem cell factor, FLT-3 ligand, and megakaryocyte growth and development factor. Blood 92 (1998), 1878–1886.
-
(1998)
Blood
, vol.92
, pp. 1878-1886
-
-
Kiem, H.P.1
Andrews, R.G.2
Morris, J.3
Peterson, L.4
Heyward, S.5
Allen, J.M.6
Rasko, J.E.7
Potter, J.8
Miller, A.D.9
-
15
-
-
0034153385
-
+ progenitors using clinically feasible methods
-
+ progenitors using clinically feasible methods. Mol Ther 1 (2000), 285–293.
-
(2000)
Mol Ther
, vol.1
, pp. 285-293
-
-
Wu, T.1
Kim, H.J.2
Sellers, S.E.3
Meade, K.E.4
Agricola, B.A.5
Metzger, M.E.6
Kato, I.7
Donahue, R.E.8
Dunbar, C.E.9
Tisdale, J.F.10
-
16
-
-
16044366110
-
Colocalization of retrovirus and target cells on specific fibronectin fragments increases genetic transduction of mammalian cells
-
16 Hanenberg, H., Xiao, X.L., Diloo, D., Hashino, K., Kato, I., Williams, D.A., Colocalization of retrovirus and target cells on specific fibronectin fragments increases genetic transduction of mammalian cells. Nat Med 2 (1996), 876–882.
-
(1996)
Nat Med
, vol.2
, pp. 876-882
-
-
Hanenberg, H.1
Xiao, X.L.2
Diloo, D.3
Hashino, K.4
Kato, I.5
Williams, D.A.6
-
17
-
-
0032079434
-
Growth-supporting activities of fibronectin on hematopoietic stem/progenitor cells in vitro and in vivo: structural requirement for fibronectin activities of CS1 and cell-binding domains
-
17 Yokota, T., Oritani, K., Mitsui, H., Aoyama, K., Ishikawa, J., Sugahara, H., Matsumura, I., Tsai, S., Tomiyama, Y., Kanakura, Y., Matsuzawa, Y., Growth-supporting activities of fibronectin on hematopoietic stem/progenitor cells in vitro and in vivo: structural requirement for fibronectin activities of CS1 and cell-binding domains. Blood 91 (1998), 3263–3272.
-
(1998)
Blood
, vol.91
, pp. 3263-3272
-
-
Yokota, T.1
Oritani, K.2
Mitsui, H.3
Aoyama, K.4
Ishikawa, J.5
Sugahara, H.6
Matsumura, I.7
Tsai, S.8
Tomiyama, Y.9
Kanakura, Y.10
Matsuzawa, Y.11
-
18
-
-
27144518462
-
Dose-dependent effects of the Notch ligand Delta1 on ex vivo differentiation and in vivo marrow repopulating ability of cord blood cells
-
18 Delaney, C., Varnum-Finney, B., Aoyama, K., Brashem-Stein, C., Berstein, I.D., Dose-dependent effects of the Notch ligand Delta1 on ex vivo differentiation and in vivo marrow repopulating ability of cord blood cells. Blood 106 (2005), 2693–2699.
-
(2005)
Blood
, vol.106
, pp. 2693-2699
-
-
Delaney, C.1
Varnum-Finney, B.2
Aoyama, K.3
Brashem-Stein, C.4
Berstein, I.D.5
-
19
-
-
84905745710
-
Rapamycin relieves lentiviral vector transduction resistance in human and mouse hematopoietic stem cells
-
Using the small molecule drug rapamycin can be used to increase transduction efficiency ex vivo without decreasing engraftment potential of hematopoietic stem cells.
-
19•• Wang, C.X., Sather, B.D., Wang, X., Adair, J.E., Khan, I., Singh, S., Lang, S., Adams, A., Curinga, G., Kiem, H.P., Miao, C.H., Rapamycin relieves lentiviral vector transduction resistance in human and mouse hematopoietic stem cells. Blood 124 (2014), 913–923 Using the small molecule drug rapamycin can be used to increase transduction efficiency ex vivo without decreasing engraftment potential of hematopoietic stem cells.
-
(2014)
Blood
, vol.124
, pp. 913-923
-
-
Wang, C.X.1
Sather, B.D.2
Wang, X.3
Adair, J.E.4
Khan, I.5
Singh, S.6
Lang, S.7
Adams, A.8
Curinga, G.9
Kiem, H.P.10
Miao, C.H.11
-
20
-
-
77956551196
-
Systematic comparison of constitutive promoters and the Doxycycline-inducible promoter
-
20 Qin, J.Y., Zhang, L., Clift, K.L., Hulur, I., Xiang, A.P., Ren, B.Z., Lahn, B.T., Systematic comparison of constitutive promoters and the Doxycycline-inducible promoter. PLoS One, 5, 2010, e10611.
-
(2010)
PLoS One
, vol.5
, pp. e10611
-
-
Qin, J.Y.1
Zhang, L.2
Clift, K.L.3
Hulur, I.4
Xiang, A.P.5
Ren, B.Z.6
Lahn, B.T.7
-
21
-
-
84989236346
-
Pgk-mediated expression of common gamma chain Is more effective than EF1a for therapeutic immune reconstitution of X-SCID dogs after in vivo gene therapy with foamy virus vector
-
21 Humbert, O., Burtner, C.R., O'Donnell, P., Humphry, D.R., Hubbard, N., Chan, F., Adair, J.E., Trobridge, G.D., Torgerson, T., Scharenberg, A.M., Rawlings, D.J., Pgk-mediated expression of common gamma chain Is more effective than EF1a for therapeutic immune reconstitution of X-SCID dogs after in vivo gene therapy with foamy virus vector. Blood, 126, 2015, 262.
-
(2015)
Blood
, vol.126
, pp. 262
-
-
Humbert, O.1
Burtner, C.R.2
O'Donnell, P.3
Humphry, D.R.4
Hubbard, N.5
Chan, F.6
Adair, J.E.7
Trobridge, G.D.8
Torgerson, T.9
Scharenberg, A.M.10
Rawlings, D.J.11
-
22
-
-
84879867061
-
Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome
-
22 Aiuti, A., Biasco, L., Scaramuzza, S., Ferrua, F., Cicalese, M.P., Baricordi, C., Dionisio, F., Calabria, A., Giannelli, S., Castiello, M.C., Bosticardo, M., Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome. Science, 341, 2013, 1233151.
-
(2013)
Science
, vol.341
, pp. 1233151
-
-
Aiuti, A.1
Biasco, L.2
Scaramuzza, S.3
Ferrua, F.4
Cicalese, M.P.5
Baricordi, C.6
Dionisio, F.7
Calabria, A.8
Giannelli, S.9
Castiello, M.C.10
Bosticardo, M.11
-
23
-
-
33646564701
-
Endogenous microRNA regulation suppresses transgene expression in hematopoietic lineages and enables stable gene transfer
-
23 Brown, B.D., Venneri, M.A., Zingale, A., Sergi Sergi, L., Naldini, L., Endogenous microRNA regulation suppresses transgene expression in hematopoietic lineages and enables stable gene transfer. Nat Med 12 (2006), 585–591.
-
(2006)
Nat Med
, vol.12
, pp. 585-591
-
-
Brown, B.D.1
Venneri, M.A.2
Zingale, A.3
Sergi Sergi, L.4
Naldini, L.5
-
24
-
-
36849060584
-
Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state
-
24 Brown, B.D., Gentner, B., Cantore, A., Colleoni, S., Amendola, M., Zingale, A., Baccarini, A., Lazzari, G., Galli, C., Naldini, L., Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state. Nat Biotechnol 25 (2007), 1457–1467.
-
(2007)
Nat Biotechnol
, vol.25
, pp. 1457-1467
-
-
Brown, B.D.1
Gentner, B.2
Cantore, A.3
Colleoni, S.4
Amendola, M.5
Zingale, A.6
Baccarini, A.7
Lazzari, G.8
Galli, C.9
Naldini, L.10
-
26
-
-
77149126435
-
A genetic strategy for single and combinatorial analysis of miRNA function in mammalian hematopoietic stem cells
-
26 Papapetrou, E.P., Korkola, J.E., Sadelain, M., A genetic strategy for single and combinatorial analysis of miRNA function in mammalian hematopoietic stem cells. Stem Cells 28 (2010), 287–296.
-
(2010)
Stem Cells
, vol.28
, pp. 287-296
-
-
Papapetrou, E.P.1
Korkola, J.E.2
Sadelain, M.3
-
27
-
-
61749100637
-
Harnessing endogenous miR-181a to segregate transgenic antigen receptor expression in developing versus post-thymic T cells in murine hematopoietic chimeras
-
27 Papapetrou, E.P., Kovalovsky, D., Beloeil, L., Sant'angelo, D., Sadelain, M., Harnessing endogenous miR-181a to segregate transgenic antigen receptor expression in developing versus post-thymic T cells in murine hematopoietic chimeras. J Clin Invest 119 (2009), 157–168.
-
(2009)
J Clin Invest
, vol.119
, pp. 157-168
-
-
Papapetrou, E.P.1
Kovalovsky, D.2
Beloeil, L.3
Sant'angelo, D.4
Sadelain, M.5
-
28
-
-
78449270317
-
Identification of hematopoietic stem cell-specific miRNAs enables gene therapy of globoid cell leukodystrophy
-
58ra84
-
28 Gentner, B., Visigalli, I., Hiramatsu, H., Lechman, E., Ungari, S., Giustacchini, A., Schira, G., Amendola, M., Quattrini, A., Martino, S., Orlacchio, A., Dick, J.E., Biffi, A., Naldini, L., Identification of hematopoietic stem cell-specific miRNAs enables gene therapy of globoid cell leukodystrophy. Sci Transl Med, 2, 2010 58ra84.
-
(2010)
Sci Transl Med
, vol.2
-
-
Gentner, B.1
Visigalli, I.2
Hiramatsu, H.3
Lechman, E.4
Ungari, S.5
Giustacchini, A.6
Schira, G.7
Amendola, M.8
Quattrini, A.9
Martino, S.10
Orlacchio, A.11
Dick, J.E.12
Biffi, A.13
Naldini, L.14
-
29
-
-
36849053006
-
A microRNA-regulated lentiviral vector mediates stable correction of hemophilia B mice
-
29 Brown, B.D., Cantore, A., Annoni, A., Sergi Sergi, L., Lombardo, A., Valle, P.D., D'Angelo, A., Naldini, L., A microRNA-regulated lentiviral vector mediates stable correction of hemophilia B mice. Blood 110 (2007), 4144–4152.
-
(2007)
Blood
, vol.110
, pp. 4144-4152
-
-
Brown, B.D.1
Cantore, A.2
Annoni, A.3
Sergi Sergi, L.4
Lombardo, A.5
Valle, P.D.6
D'Angelo, A.7
Naldini, L.8
-
30
-
-
84905451458
-
Dual-regulated lentiviral vector for gene therapy of X-linked Chronic Granulomatosis
-
The authors developed a dual regulated lentiviral vector that restored up to 50% NADPH activity in X-CGD mice and primary human phagocytes from X-CGD patients. Using a myeloid specific promoter and miR-126, expression of the therapeutic gene was targeted specifically to the myeloid lineage and de-targeted expression from HSCs.
-
30•• Chiriaco, M., Farinelli, G., Capo, V., Zonari, E., Scaramuzza, S., Di Matteo, G., Sergi Sergi, L., Migliavacca, M., Hernandez, R.J., Bombelli, F., Giorda, E., Dual-regulated lentiviral vector for gene therapy of X-linked Chronic Granulomatosis. Mol Ther 22 (2014), 1472–1483 The authors developed a dual regulated lentiviral vector that restored up to 50% NADPH activity in X-CGD mice and primary human phagocytes from X-CGD patients. Using a myeloid specific promoter and miR-126, expression of the therapeutic gene was targeted specifically to the myeloid lineage and de-targeted expression from HSCs.
-
(2014)
Mol Ther
, vol.22
, pp. 1472-1483
-
-
Chiriaco, M.1
Farinelli, G.2
Capo, V.3
Zonari, E.4
Scaramuzza, S.5
Di Matteo, G.6
Sergi Sergi, L.7
Migliavacca, M.8
Hernandez, R.J.9
Bombelli, F.10
Giorda, E.11
-
31
-
-
33646887376
-
Genotoxicity of retroviral integration in hematopoietic cells
-
31 Nienhuis, A.W., Dunbar, C.E., Sorrentino, B.P., Genotoxicity of retroviral integration in hematopoietic cells. Mol Ther 13 (2006), 1031–1047.
-
(2006)
Mol Ther
, vol.13
, pp. 1031-1047
-
-
Nienhuis, A.W.1
Dunbar, C.E.2
Sorrentino, B.P.3
-
32
-
-
21044448037
-
Clonal dominance of hematopoietic stem cells triggered by retroviral gene marking
-
32 Kustikova, O., Fehse, B., Modlich, U., Yang, M., Dullmann, J., Kamino, K., Clonal dominance of hematopoietic stem cells triggered by retroviral gene marking. Science 308 (2005), 1171–1174.
-
(2005)
Science
, vol.308
, pp. 1171-1174
-
-
Kustikova, O.1
Fehse, B.2
Modlich, U.3
Yang, M.4
Dullmann, J.5
Kamino, K.6
-
33
-
-
70449127230
-
Insertional transformation of hematopoietic cells by self-inactivating lentiviral and gammaretroviral vectors
-
33 Modlich, U., Navarro, S., Zychlinski, D., Maetzig, T., Knoess, S., Brugman, M.H., Insertional transformation of hematopoietic cells by self-inactivating lentiviral and gammaretroviral vectors. Mol Ther 17 (2009), 1919–1928.
-
(2009)
Mol Ther
, vol.17
, pp. 1919-1928
-
-
Modlich, U.1
Navarro, S.2
Zychlinski, D.3
Maetzig, T.4
Knoess, S.5
Brugman, M.H.6
-
34
-
-
51349158298
-
Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1
-
34 Howe, S.J., Mansour, M.R., Schwarzwaelder, K., Bartholomae, C., Hubank, M., Kempski, H., Brugman, M.H., Pike-Overzet, K., Chatters, S.J., de Ridder, D., Gilmour, K.C., Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1. J Clin Invest 118 (2008), 3143–3150.
-
(2008)
J Clin Invest
, vol.118
, pp. 3143-3150
-
-
Howe, S.J.1
Mansour, M.R.2
Schwarzwaelder, K.3
Bartholomae, C.4
Hubank, M.5
Kempski, H.6
Brugman, M.H.7
Pike-Overzet, K.8
Chatters, S.J.9
de Ridder, D.10
Gilmour, K.C.11
-
35
-
-
0142084745
-
LMO2-associated clonal T-cell proliferation in two patients after gene therapy for SCID-X1
-
35 Hacein-Bey-Abina, S., von Kalle, C., Schmidt, M., McCormack, M.P., Wulffraat, N., Leboulch, P., Lim, A., Osborne, C.S., Pawliuk, R., Morillon, E., Sorenson, R., LMO2-associated clonal T-cell proliferation in two patients after gene therapy for SCID-X1. Science 302 (2003), 415–419.
-
(2003)
Science
, vol.302
, pp. 415-419
-
-
Hacein-Bey-Abina, S.1
von Kalle, C.2
Schmidt, M.3
McCormack, M.P.4
Wulffraat, N.5
Leboulch, P.6
Lim, A.7
Osborne, C.S.8
Pawliuk, R.9
Morillon, E.10
Sorenson, R.11
-
36
-
-
0037448352
-
A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency
-
36 Hacein-Bey-Abina, S., von Kalle, C., Schmidt, M., Le Deist, F., Wulffraat, N., McIntyre, E., Radford, I., Villeval, J.L., Fraser, C.C., Cavazzana-Calvo, M., Fischer, A., A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N Engl J Med 348 (2003), 255–256.
-
(2003)
N 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
Radford, I.7
Villeval, J.L.8
Fraser, C.C.9
Cavazzana-Calvo, M.10
Fischer, A.11
-
37
-
-
51349090473
-
Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1
-
37 Hacein-Bey-Abina, S., Garrigue, A., Wang, G.P., Soulier, J., Lim, A., Morillon, E., Clappier, E., Caccavelli, L., Delabesse, E., Beldjord, K., Asnafi, V., Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. J Clin Invest 118 (2008), 3132–3142.
-
(2008)
J Clin Invest
, vol.118
, pp. 3132-3142
-
-
Hacein-Bey-Abina, S.1
Garrigue, A.2
Wang, G.P.3
Soulier, J.4
Lim, A.5
Morillon, E.6
Clappier, E.7
Caccavelli, L.8
Delabesse, E.9
Beldjord, K.10
Asnafi, V.11
-
38
-
-
0037841763
-
Transcription start regions in the human genome are favored targets for MLV integration
-
38 Wu, X., Li, Y., Crise, B., Burgess, S.M., Transcription start regions in the human genome are favored targets for MLV integration. Science 300 (2003), 1749–1751.
-
(2003)
Science
, vol.300
, pp. 1749-1751
-
-
Wu, X.1
Li, Y.2
Crise, B.3
Burgess, S.M.4
-
39
-
-
0037162715
-
HIV-1 integration in the human genome favors active genes and local hotspots
-
39 Schroeder, A.R., Shinn, P., Chen, H., Berry, C., Ecker, J.R., Bushman, F., HIV-1 integration in the human genome favors active genes and local hotspots. Cell 110 (2002), 521–529.
-
(2002)
Cell
, vol.110
, pp. 521-529
-
-
Schroeder, A.R.1
Shinn, P.2
Chen, H.3
Berry, C.4
Ecker, J.R.5
Bushman, F.6
-
40
-
-
34250851635
-
Comparison of HIV-derived lentiviral and MLV-based gammaretroviral vector integration sites in primate repopulating cells
-
40 Beard, B.C., Dickerson, D., Beebe, K., Gooch, C., Fletcher, J., Okbinoglu, Miller, D.G., Jacobs, M.A., Kiem, H.P., Trobridge, G.D., Comparison of HIV-derived lentiviral and MLV-based gammaretroviral vector integration sites in primate repopulating cells. Mol Ther 15 (2007), 1356–1365.
-
(2007)
Mol Ther
, vol.15
, pp. 1356-1365
-
-
Beard, B.C.1
Dickerson, D.2
Beebe, K.3
Gooch, C.4
Fletcher, J.5
Okbinoglu6
Miller, D.G.7
Jacobs, M.A.8
Kiem, H.P.9
Trobridge, G.D.10
-
41
-
-
34249804819
-
Unique integration profiles in canine model of long-term repopulating cells transduced with gammaretrovirus, lentivirus, or foamy virus
-
41 Beard, B.C., Keyser, K.A., Trobridge, G.D., Peterson, L.J., Miller, D.G., Jacobs, M., Kaul, R., Kiem, H.P., Unique integration profiles in canine model of long-term repopulating cells transduced with gammaretrovirus, lentivirus, or foamy virus. Hum Gene Ther 18 (2007), 423–434.
-
(2007)
Hum Gene Ther
, vol.18
, pp. 423-434
-
-
Beard, B.C.1
Keyser, K.A.2
Trobridge, G.D.3
Peterson, L.J.4
Miller, D.G.5
Jacobs, M.6
Kaul, R.7
Kiem, H.P.8
-
42
-
-
31944444349
-
Foamy virus vector integration sites in normal human cells
-
42 Trobridge, G.D., Miller, D.G., Jacobs, M.A., Allen, J.M., Kiem, H.P., Kaul, R., Russell, D.W., Foamy virus vector integration sites in normal human cells. Proc Natl Acad Sci USA 103 (2006), 1498–1503.
-
(2006)
Proc Natl Acad Sci USA
, vol.103
, pp. 1498-1503
-
-
Trobridge, G.D.1
Miller, D.G.2
Jacobs, M.A.3
Allen, J.M.4
Kiem, H.P.5
Kaul, R.6
Russell, D.W.7
-
44
-
-
33745684269
-
Retroviral DNA integration: viral and cellular determinants of target-site selection
-
44 Lewinski, M.K., Yamashita, M., Emerman, M., Ciuffi, A., Marshall, H., Crawford, G., Collins, F., Shinn, P., Leipzig, J., Hannenhalli, S., Berry, C.C., Ecker, J.R., Bushman, F.D., Retroviral DNA integration: viral and cellular determinants of target-site selection. PLoS Pathog, 2, 2006, e60.
-
(2006)
PLoS Pathog
, vol.2
, pp. e60
-
-
Lewinski, M.K.1
Yamashita, M.2
Emerman, M.3
Ciuffi, A.4
Marshall, H.5
Crawford, G.6
Collins, F.7
Shinn, P.8
Leipzig, J.9
Hannenhalli, S.10
Berry, C.C.11
Ecker, J.R.12
Bushman, F.D.13
-
45
-
-
77649250416
-
LEDGF hybrids efficiently retarget lentiviral integration into heterochromatin
-
45 Gijsbers, R., Ronen, K., Vets, S., Malani, N., De Rijck, J., McNeely, M., Bushman, F.D., Debyser, Z., LEDGF hybrids efficiently retarget lentiviral integration into heterochromatin. Mol Ther 18 (2010), 552–560.
-
(2010)
Mol Ther
, vol.18
, pp. 552-560
-
-
Gijsbers, R.1
Ronen, K.2
Vets, S.3
Malani, N.4
De Rijck, J.5
McNeely, M.6
Bushman, F.D.7
Debyser, Z.8
-
46
-
-
0347634423
-
Fusion proteins consisting of human immunodeficiency virus type 1 integrase and the designed polydactyl zinc finger protein E2C direct integration of viral DNA into specific sites
-
46 Tan, W., Zhu, K., Segal, D.J., Barbas, C.F., Chow, S.A., Fusion proteins consisting of human immunodeficiency virus type 1 integrase and the designed polydactyl zinc finger protein E2C direct integration of viral DNA into specific sites. J Virol 78 (2004), 1301–1313.
-
(2004)
J Virol
, vol.78
, pp. 1301-1313
-
-
Tan, W.1
Zhu, K.2
Segal, D.J.3
Barbas, C.F.4
Chow, S.A.5
-
47
-
-
84876359026
-
rDNA-directed integration by an HIV-1 integrase-I-Pol fusion protein
-
47 Schenkwein, D., Turkki, V., Ahlroth, M.K., Timonen, O., Airenne, K.J., Yla-Herttuala, S., rDNA-directed integration by an HIV-1 integrase-I-Pol fusion protein. Nucl Acids Res, 41, 2013, e61.
-
(2013)
Nucl Acids Res
, vol.41
, pp. e61
-
-
Schenkwein, D.1
Turkki, V.2
Ahlroth, M.K.3
Timonen, O.4
Airenne, K.J.5
Yla-Herttuala, S.6
-
48
-
-
84905219960
-
BET-independent MLV-based vectors target away from promoters and regulatory elements
-
By introducing mutations into BET proteins, gammaretroviral vectors are targeted away from actively transcribed regions of genes (including proto-oncogenes) in NIH3T3 and HeLa cells.
-
48• El Ashkar, S., De Rijck, J., Demeulemeester, J., Vets, S., Madlala, P., Cermakova, K., Debyser, Z., Gijsbers, R., BET-independent MLV-based vectors target away from promoters and regulatory elements. Mol Ther Nucl Acids, 3, 2014, e179 By introducing mutations into BET proteins, gammaretroviral vectors are targeted away from actively transcribed regions of genes (including proto-oncogenes) in NIH3T3 and HeLa cells.
-
(2014)
Mol Ther Nucl Acids
, vol.3
, pp. e179
-
-
El Ashkar, S.1
De Rijck, J.2
Demeulemeester, J.3
Vets, S.4
Madlala, P.5
Cermakova, K.6
Debyser, Z.7
Gijsbers, R.8
-
49
-
-
0033601096
-
Putting boundaries on silence
-
49 Sun, F.L., Elgin, S.C., Putting boundaries on silence. Cell 99 (1999), 459–462.
-
(1999)
Cell
, vol.99
, pp. 459-462
-
-
Sun, F.L.1
Elgin, S.C.2
-
50
-
-
84884825207
-
Identification and characterization of enhancer-blocking insulators to reduce retroviral genotoxicity
-
50 Groth, A.C., Liu, M., Wang, H., Lovelett, E., Emery, D.W., Identification and characterization of enhancer-blocking insulators to reduce retroviral genotoxicity. PLoS One, 8, 2013, e76528.
-
(2013)
PLoS One
, vol.8
, pp. e76528
-
-
Groth, A.C.1
Liu, M.2
Wang, H.3
Lovelett, E.4
Emery, D.W.5
-
51
-
-
84923278709
-
Genomic discovery of potent chromatin insulators for human gene therapy
-
The authors discovered and characterized multiple novel chromatin insulators that provide enhancer-blocking or barrier activity. Some of these insulators appear to have more enhancer-blocking activity than the commonly used cHS4 insulator.
-
51•• Liu, M., Maurano, M.T., Wang, H., Qi, H., Song, C.Z., Navas, P.A., Emery, D.W., Stamatoyannopoulos, J.A., Stamatoyannopoulos, G., Genomic discovery of potent chromatin insulators for human gene therapy. Nat Biotechnol 33 (2015), 198–203 The authors discovered and characterized multiple novel chromatin insulators that provide enhancer-blocking or barrier activity. Some of these insulators appear to have more enhancer-blocking activity than the commonly used cHS4 insulator.
-
(2015)
Nat Biotechnol
, vol.33
, pp. 198-203
-
-
Liu, M.1
Maurano, M.T.2
Wang, H.3
Qi, H.4
Song, C.Z.5
Navas, P.A.6
Emery, D.W.7
Stamatoyannopoulos, J.A.8
Stamatoyannopoulos, G.9
-
52
-
-
84962050350
-
Insulated foamy viral vectors
-
+ cells. The authors also developed a rapid and sensitive plasmid-based assay for testing insulator function.
-
+ cells. The authors also developed a rapid and sensitive plasmid-based assay for testing insulator function.
-
(2015)
Hum Gene Ther
, vol.27
, pp. 255-266
-
-
Browning, D.1
Collins, C.2
Leap, D.3
Hocum, J.4
Trobridge, G.D.5
-
53
-
-
63949083133
-
Genomic and functional assays demonstrate reduced gammaretroviral genotoxicity associated with use of the cHS4 chromatin insulator
-
53 Li Cl, Xiong, D., Stamatoyannopoulos, G., Emery, D.W., Genomic and functional assays demonstrate reduced gammaretroviral genotoxicity associated with use of the cHS4 chromatin insulator. Mol Ther 17 (2009), 716–724.
-
(2009)
Mol Ther
, vol.17
, pp. 716-724
-
-
Li Cl1
Xiong, D.2
Stamatoyannopoulos, G.3
Emery, D.W.4
-
54
-
-
84897570147
-
Uncovering and dissecting the genotoxicity of self-inactivating lentiviral vectors in vivo
-
54 Cesana, D., Ranzani, M., Volpin, M., Bartholomae, C., Duros, C., Artus, A., Merella, S., Benedicenti, F., Sergi Sergi, L., Sanvito, F., Uncovering and dissecting the genotoxicity of self-inactivating lentiviral vectors in vivo. Mol Ther 22 (2014), 774–785.
-
(2014)
Mol Ther
, vol.22
, pp. 774-785
-
-
Cesana, D.1
Ranzani, M.2
Volpin, M.3
Bartholomae, C.4
Duros, C.5
Artus, A.6
Merella, S.7
Benedicenti, F.8
Sergi Sergi, L.9
Sanvito, F.10
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