-
1
-
-
0142084745
-
LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1
-
S. Hacein-Bey-Abina, C. Kalle Von, M. Schmidt, M.P. McCormack, N. Wulffraat, P. Leboulch, A. Lim, C.S. Osborne, R. Pawliuk, E. Morillon, and et al. 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
-
2
-
-
51349158298
-
Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients
-
S.J. Howe, M.R. Mansour, K. Schwarzwaelder, C. Bartholomae, M. Hubank, H. Kempski, M.H. Brugman, K. Pike-Overzet, S.J. Chatters, D. de Ridder, and et al. Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients J Clin Investig 118 2008 3143 3150
-
(2008)
J Clin Investig
, 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
-
3
-
-
76249131912
-
Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease
-
S. Stein, M.G. Ott, S. Schultze-Strasser, A. Jauch, B. Burwinkel, A. Kinner, M. Schmidt, A. Krämer, J. Schwäble, H. Glimm, and et al. Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease Nat Med 16 2010 198 204
-
(2010)
Nat Med
, vol.16
, pp. 198-204
-
-
Stein, S.1
Ott, M.G.2
Schultze-Strasser, S.3
Jauch, A.4
Burwinkel, B.5
Kinner, A.6
Schmidt, M.7
Krämer, A.8
Schwäble, J.9
Glimm, H.10
-
4
-
-
84907294964
-
Gene therapy for Wiskott-Aldrich syndrome - Long-term efficacy and genotoxicity
-
C.J. Braun, K. Boztug, A. Paruzynski, M. Witzel, A. Schwarzer, M. Rothe, U. Modlich, R. Beier, G. Göhring, D. Steinemann, and et al. Gene therapy for Wiskott-Aldrich syndrome - long-term efficacy and genotoxicity Sci Transl Med 6 2014 227ra33
-
(2014)
Sci Transl Med
, vol.6
, pp. 227ra33
-
-
Braun, C.J.1
Boztug, K.2
Paruzynski, A.3
Witzel, M.4
Schwarzer, A.5
Rothe, M.6
Modlich, U.7
Beier, R.8
Göhring, G.9
Steinemann, D.10
-
5
-
-
0346132122
-
Self-inactivating retroviral vectors designed for transfer of whole genes into mammalian cells
-
S.F. Yu, T. Rüden von, P.W. Kantoff, C. Garber, M. Seiberg, U. Rüther, W.F. Anderson, E.F. Wagner, and E. Gilboa Self-inactivating retroviral vectors designed for transfer of whole genes into mammalian cells Proc Natl Acad Sci U S A 83 1986 3194 3198
-
(1986)
Proc Natl Acad Sci U S A
, vol.83
, pp. 3194-3198
-
-
Yu, S.F.1
Rüden Von, T.2
Kantoff, P.W.3
Garber, C.4
Seiberg, M.5
Rüther, U.6
Anderson, W.F.7
Wagner, E.F.8
Gilboa, E.9
-
6
-
-
7244231295
-
Self-inactivating retroviral vectors with improved RNA processing
-
J. Kraunus, D.H.S. Schaumann, J. Meyer, U. Modlich, B. Fehse, G. Brandenburg, D. Laer von, H. Klump, A. Schambach, J. Bohne, and et al. Self-inactivating retroviral vectors with improved RNA processing Gene Ther 11 2004 1568 1578
-
(2004)
Gene Ther
, vol.11
, pp. 1568-1578
-
-
Kraunus, J.1
Schaumann, D.H.S.2
Meyer, J.3
Modlich, U.4
Fehse, B.5
Brandenburg, G.6
Laer Von, D.7
Klump, H.8
Schambach, A.9
Bohne, J.10
-
7
-
-
0031743608
-
Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery
-
R. Zufferey, T. Dull, R.J. Mandel, A. Bukovsky, D. Quiroz, L. Naldini, and D. Trono Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery J Virol 72 1998 9873 9880
-
(1998)
J Virol
, vol.72
, pp. 9873-9880
-
-
Zufferey, R.1
Dull, T.2
Mandel, R.J.3
Bukovsky, A.4
Quiroz, D.5
Naldini, L.6
Trono, D.7
-
8
-
-
0037841763
-
Transcription start regions in the human genome are favored targets for MLV integration
-
X. Wu, Y. Li, B. Crise, and S.M. Burgess 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
-
9
-
-
34547657665
-
Hot spots of retroviral integration in human CD34+ hematopoietic cells
-
C. Cattoglio, G. Facchini, D. Sartori, A. Antonelli, A. Miccio, B. Cassani, M. Schmidt, C. Kalle Von, S. Howe, A.J. Thrasher, and et al. Hot spots of retroviral integration in human CD34+ hematopoietic cells Blood 110 2007 1770 1778
-
(2007)
Blood
, vol.110
, pp. 1770-1778
-
-
Cattoglio, C.1
Facchini, G.2
Sartori, D.3
Antonelli, A.4
Miccio, A.5
Cassani, B.6
Schmidt, M.7
Kalle Von, C.8
Howe, S.9
Thrasher, A.J.10
-
10
-
-
79954733266
-
Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection
-
A. Biffi, C.C. Bartolomae, D. Cesana, N. Cartier, P. Aubourg, M. Ranzani, M. Cesani, F. Benedicenti, T. Plati, E. Rubagotti, and et al. Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection Blood 117 2011 5332 5339
-
(2011)
Blood
, vol.117
, pp. 5332-5339
-
-
Biffi, A.1
Bartolomae, C.C.2
Cesana, D.3
Cartier, N.4
Aubourg, P.5
Ranzani, M.6
Cesani, M.7
Benedicenti, F.8
Plati, T.9
Rubagotti, E.10
-
11
-
-
0028069577
-
Severe combined immunodeficiency, interleukin-2 (IL-2), and the IL-2 receptor: Experiments of nature continue to point the way
-
S.D. Voss, R. Hong, and P.M. Sondel Severe combined immunodeficiency, interleukin-2 (IL-2), and the IL-2 receptor: experiments of nature continue to point the way Blood 83 1994 626 635
-
(1994)
Blood
, vol.83
, pp. 626-635
-
-
Voss, S.D.1
Hong, R.2
Sondel, P.M.3
-
12
-
-
39849110286
-
Self-inactivating gammaretroviral vectors for gene therapy of X-linked severe combined immunodeficiency
-
S.I. Thornhill, A. Schambach, S.J. Howe, M. Ulaganathan, E. Grassman, D. Williams, B. Schiedlmeier, N.I.J. Sebire, H.B. Gaspar, C. Kinnon, and et al. Self-inactivating gammaretroviral vectors for gene therapy of X-linked severe combined immunodeficiency Mol Ther 16 2008 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
Schiedlmeier, B.7
Sebire, N.I.J.8
Gaspar, H.B.9
Kinnon, C.10
-
13
-
-
41149133413
-
Physiological promoters reduce the genotoxic risk of integrating gene vectors
-
D. Zychlinski, A. Schambach, U. Modlich, T. Maetzig, J. Meyer, E. Grassman, A. Mishra, and C. Baum Physiological promoters reduce the genotoxic risk of integrating gene vectors Mol Ther 16 2008 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
Mishra, A.7
Baum, C.8
-
14
-
-
84907880404
-
A modified γ-retrovirus vector for X-linked severe combined immunodeficiency
-
S. Hacein-Bey Abina, S.-Y. Pai, H.B. Gaspar, M. Armant, C.C. Berry, S. Blanche, J. Bleesing, J. Blondeau, H. de Boer, K.F. Buckland, and et al. A modified γ-retrovirus vector for X-linked severe combined immunodeficiency N Engl J Med 371 2014 1407 1417
-
(2014)
N Engl J Med
, vol.371
, pp. 1407-1417
-
-
Hacein-Bey Abina, S.1
Pai, S.-Y.2
Gaspar, H.B.3
Armant, M.4
Berry, C.C.5
Blanche, S.6
Bleesing, J.7
Blondeau, J.8
De Boer, H.9
Buckland, K.F.10
-
15
-
-
0034968336
-
Cognitive and behavioral abnormalities in adenosine deaminase deficient severe combined immunodeficiency
-
M.H. Rogers, R. Lwin, L. Fairbanks, B. Gerritsen, and H.B. Gaspar Cognitive and behavioral abnormalities in adenosine deaminase deficient severe combined immunodeficiency J Pediatr 139 2001 44 50
-
(2001)
J Pediatr
, vol.139
, pp. 44-50
-
-
Rogers, M.H.1
Lwin, R.2
Fairbanks, L.3
Gerritsen, B.4
Gaspar, H.B.5
-
16
-
-
55749109027
-
Cognitive and behavioral abnormalities in children after hematopoietic stem cell transplantation for severe congenital immunodeficiencies
-
P. Titman, E. Pink, E. Skucek, K. O'Hanlon, T.J. Cole, J. Gaspar, J. Xu-Bayford, A. Jones, A.J. Thrasher, E.G. Davies, and et al. Cognitive and behavioral abnormalities in children after hematopoietic stem cell transplantation for severe congenital immunodeficiencies Blood 112 2008 3907 3913
-
(2008)
Blood
, vol.112
, pp. 3907-3913
-
-
Titman, P.1
Pink, E.2
Skucek, E.3
O'Hanlon, K.4
Cole, T.J.5
Gaspar, J.6
Xu-Bayford, J.7
Jones, A.8
Thrasher, A.J.9
Davies, E.G.10
-
17
-
-
34547686397
-
Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy
-
A. Aiuti, B. Cassani, G. Andolfi, M. Mirolo, L. Biasco, A. Recchia, F. Urbinati, C. Valacca, S. Scaramuzza, M. Aker, and et al. Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy J Clin Investig 117 2007 2233 2240
-
(2007)
J Clin Investig
, vol.117
, pp. 2233-2240
-
-
Aiuti, A.1
Cassani, B.2
Andolfi, G.3
Mirolo, M.4
Biasco, L.5
Recchia, A.6
Urbinati, F.7
Valacca, C.8
Scaramuzza, S.9
Aker, M.10
-
18
-
-
84878189027
-
Wiskott-Aldrich syndrome: A comprehensive review
-
M.J. Massaad, N. Ramesh, and R.S. Geha Wiskott-Aldrich syndrome: a comprehensive review Ann N Y Acad Sci 1285 2013 26 43
-
(2013)
Ann N y Acad Sci
, vol.1285
, pp. 26-43
-
-
Massaad, M.J.1
Ramesh, N.2
Geha, R.S.3
-
19
-
-
0031839717
-
A 5' regulatory sequence containing two Ets motifs controls the expression of the Wiskott-Aldrich syndrome protein (WASP) gene in human hematopoietic cells
-
A. Petrella, I. Doti, V. Agosti, P.C. Giarrusso, D. Vitale, H.M. Bond, C. Cuomo, P. Tassone, B. Franco, A. Ballabio, and et al. A 5' regulatory sequence containing two Ets motifs controls the expression of the Wiskott-Aldrich syndrome protein (WASP) gene in human hematopoietic cells Blood 91 1998 4554 4560
-
(1998)
Blood
, vol.91
, pp. 4554-4560
-
-
Petrella, A.1
Doti, I.2
Agosti, V.3
Giarrusso, P.C.4
Vitale, D.5
Bond, H.M.6
Cuomo, C.7
Tassone, P.8
Franco, B.9
Ballabio, A.10
-
20
-
-
33847178027
-
Lentiviral vectors targeting WASp expression to hematopoietic cells, efficiently transduce and correct cells from WAS patients
-
S. Charrier, L. Dupré, S. Scaramuzza, L. Jeanson-Leh, M.P. Blundell, O. Danos, F. Cattaneo, A. Aiuti, R. Eckenberg, A.J. Thrasher, and et al. Lentiviral vectors targeting WASp expression to hematopoietic cells, efficiently transduce and correct cells from WAS patients Gene Ther 14 2007 415 428
-
(2007)
Gene Ther
, vol.14
, pp. 415-428
-
-
Charrier, S.1
Dupré, L.2
Scaramuzza, S.3
Jeanson-Leh, L.4
Blundell, M.P.5
Danos, O.6
Cattaneo, F.7
Aiuti, A.8
Eckenberg, R.9
Thrasher, A.J.10
-
21
-
-
67349217158
-
Evidence for long-term efficacy and safety of gene therapy for Wiskott-Aldrich syndrome in preclinical models
-
F. Marangoni, M. Bosticardo, S. Charrier, E. Draghici, M. Locci, S. Scaramuzza, C. Panaroni, M. Ponzoni, F. Sanvito, C. Doglioni, and et al. Evidence for long-term efficacy and safety of gene therapy for Wiskott-Aldrich syndrome in preclinical models Mol Ther 17 2009 1073 1082
-
(2009)
Mol Ther
, vol.17
, pp. 1073-1082
-
-
Marangoni, F.1
Bosticardo, M.2
Charrier, S.3
Draghici, E.4
Locci, M.5
Scaramuzza, S.6
Panaroni, C.7
Ponzoni, M.8
Sanvito, F.9
Doglioni, C.10
-
22
-
-
84879867061
-
Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome
-
A. Aiuti, L. Biasco, S. Scaramuzza, F. Ferrua, M.P. Cicalese, C. Baricordi, F. Dionisio, A. Calabria, S. Giannelli, M.C. Castiello, and et al. 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
-
23
-
-
84928389399
-
Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome
-
S. Hacein-Bey Abina, H.B. Gaspar, J. Blondeau, L. Caccavelli, S. Charrier, K. Buckland, C. Picard, E. Six, N. Himoudi, K. Gilmour, and et al. Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome JAMA 313 2015 1550 1563
-
(2015)
JAMA
, vol.313
, pp. 1550-1563
-
-
Hacein-Bey Abina, S.1
Gaspar, H.B.2
Blondeau, J.3
Caccavelli, L.4
Charrier, S.5
Buckland, K.6
Picard, C.7
Six, E.8
Himoudi, N.9
Gilmour, K.10
-
24
-
-
84941182289
-
B-cell reconstitution after lentiviral vector-mediated gene therapy in patients with Wiskott-Aldrich syndrome
-
M.C. Castiello, S. Scaramuzza, F. Pala, F. Ferrua, P. Uva, I. Brigida, L. Sereni, M. van der Burg, G. Ottaviano, M.H. Albert, and et al. B-cell reconstitution after lentiviral vector-mediated gene therapy in patients with Wiskott-Aldrich syndrome J Allergy Clin Immunol 2015 10.1016/j.jaci.2015.01.035
-
(2015)
J Allergy Clin Immunol
-
-
Castiello, M.C.1
Scaramuzza, S.2
Pala, F.3
Ferrua, F.4
Uva, P.5
Brigida, I.6
Sereni, L.7
Van Der Burg, M.8
Ottaviano, G.9
Albert, M.H.10
-
25
-
-
79957875084
-
Chronic granulomatous disease: Overview and hematopoietic stem cell transplantation
-
E.M. Kang, B.E. Marciano, S. DeRavin, K.A. Zarember, S.M. Holland, and H.L. Malech Chronic granulomatous disease: overview and hematopoietic stem cell transplantation J Allergy Clin Immunol 127 2011 1319 1326
-
(2011)
J Allergy Clin Immunol
, vol.127
, pp. 1319-1326
-
-
Kang, E.M.1
Marciano, B.E.2
DeRavin, S.3
Zarember, K.A.4
Holland, S.M.5
Malech, H.L.6
-
26
-
-
78650918644
-
Biochemical correction of X-CGD by a novel chimeric promoter regulating high levels of transgene expression in myeloid cells
-
G. Santilli, E. Almarza, C. Brendel, U. Choi, C. Beilin, M.P. Blundell, S. Haria, K.L. Parsley, C. Kinnon, H.L. Malech, and et al. Biochemical correction of X-CGD by a novel chimeric promoter regulating high levels of transgene expression in myeloid cells Mol Ther 19 2011 122 132
-
(2011)
Mol Ther
, vol.19
, pp. 122-132
-
-
Santilli, G.1
Almarza, E.2
Brendel, C.3
Choi, U.4
Beilin, C.5
Blundell, M.P.6
Haria, S.7
Parsley, K.L.8
Kinnon, C.9
Malech, H.L.10
-
27
-
-
84879334117
-
Human miR223 promoter as a novel myelo-specific promoter for chronic granulomatous disease gene therapy
-
C. Brendel, W. Hänseler, V. Wohlgensinger, M. Bianchi, S. Tokmak, L. Chen-Wichmann, E. Kuzmenko, N. Cesarovic, F. Nicholls, J. Reichenbach, and et al. Human miR223 promoter as a novel myelo-specific promoter for chronic granulomatous disease gene therapy Hum Gene Ther Methods 24 2013 151 159
-
(2013)
Hum Gene Ther Methods
, vol.24
, pp. 151-159
-
-
Brendel, C.1
Hänseler, W.2
Wohlgensinger, V.3
Bianchi, M.4
Tokmak, S.5
Chen-Wichmann, L.6
Kuzmenko, E.7
Cesarovic, N.8
Nicholls, F.9
Reichenbach, J.10
-
28
-
-
84905451458
-
Dual-regulated lentiviral vector for gene therapy of X-linked chronic granulomatosis
-
M. Chiriaco, G. Farinelli, V. Capo, E. Zonari, S. Scaramuzza, G. Di Matteo, L.S. Sergi, M. Migliavacca, R.J. Hernandez, F. Bombelli, and et al. Dual-regulated lentiviral vector for gene therapy of X-linked chronic granulomatosis Mol Ther 22 2014 1472 1483
-
(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, L.S.7
Migliavacca, M.8
Hernandez, R.J.9
Bombelli, F.10
-
29
-
-
0035917489
-
Artemis, a novel DNA double-strand break repair/V(D)J recombination protein, is mutated in human severe combined immune deficiency
-
D. Moshous, I. Callebaut, R. de Chasseval, B. Corneo, M. Cavazzana-Calvo, F. Le Deist, I. Tezcan, O. Sanal, Y.N P. Bertrand, N. Philippe, and et al. Artemis, a novel DNA double-strand break repair/V(D)J recombination protein, is mutated in human severe combined immune deficiency Cell 105 2001 177 186
-
(2001)
Cell
, vol.105
, pp. 177-186
-
-
Moshous, D.1
Callebaut, I.2
De Chasseval, R.3
Corneo, B.4
Cavazzana-Calvo, M.5
Le Deist, F.6
Tezcan, I.7
Sanal, O.8
Bertrand, Y.N.P.9
Philippe, N.10
-
30
-
-
33750833078
-
Complete correction of murine artemis immunodeficiency by lentiviral vector-mediated gene transfer
-
G. Mostoslavsky, A.J. Fabian, S. Rooney, F.W. Alt, and R.C. Mulligan Complete correction of murine artemis immunodeficiency by lentiviral vector-mediated gene transfer Proc Natl Acad Sci U S A 103 2006 16406 16411
-
(2006)
Proc Natl Acad Sci U S A
, vol.103
, pp. 16406-16411
-
-
Mostoslavsky, G.1
Fabian, A.J.2
Rooney, S.3
Alt, F.W.4
Mulligan, R.C.5
-
31
-
-
48349132972
-
Stable and functional lymphoid reconstitution in artemis-deficient mice following lentiviral artemis gene transfer into hematopoietic stem cells
-
F. Benjelloun, A. Garrigue, C. Demerens-de Chappedelaine, P. Soulas-Sprauel, M. Malassis-Séris, D. Stockholm, J. Hauer, J. Blondeau, J. Rivière, A. Lim, and et al. Stable and functional lymphoid reconstitution in artemis-deficient mice following lentiviral artemis gene transfer into hematopoietic stem cells Mol Ther 16 2008 1490 1499
-
(2008)
Mol Ther
, vol.16
, pp. 1490-1499
-
-
Benjelloun, F.1
Garrigue, A.2
Demerens-De Chappedelaine, C.3
Soulas-Sprauel, P.4
Malassis-Séris, M.5
Stockholm, D.6
Hauer, J.7
Blondeau, J.8
Rivière, J.9
Lim, A.10
-
32
-
-
77954906403
-
Cytotoxicity associated with artemis overexpression after lentiviral vector-mediated gene transfer
-
M. Multhaup, A.D. Karlen, D.L. Swanson, A. Wilber, N.V. Somia, M.J. Cowan, and R.S. McIvor Cytotoxicity associated with artemis overexpression after lentiviral vector-mediated gene transfer Hum Gene Ther 21 2010 865 875
-
(2010)
Hum Gene Ther
, vol.21
, pp. 865-875
-
-
Multhaup, M.1
Karlen, A.D.2
Swanson, D.L.3
Wilber, A.4
Somia, N.V.5
Cowan, M.J.6
McIvor, R.S.7
-
33
-
-
84928481340
-
Role of transgene regulation in ex vivo lentiviral correction of artemis deficiency
-
M.M. Multhaup, K.M. Podetz-Pedersen, A.D. Karlen, E.R. Olson, R. Gunther, N.V. Somia, B.R. Blazar, M.J. Cowan, and R.S. McIvor Role of transgene regulation in ex vivo lentiviral correction of artemis deficiency Hum Gene Ther 26 2015 232 243
-
(2015)
Hum Gene Ther
, vol.26
, pp. 232-243
-
-
Multhaup, M.M.1
Podetz-Pedersen, K.M.2
Karlen, A.D.3
Olson, E.R.4
Gunther, R.5
Somia, N.V.6
Blazar, B.R.7
Cowan, M.J.8
McIvor, R.S.9
-
34
-
-
33750980124
-
RAG-dependent primary immunodeficiencies
-
C. Sobacchi, V. Marrella, F. Rucci, P. Vezzoni, and A. Villa RAG-dependent primary immunodeficiencies Hum Mutat 27 2006 1174 1184
-
(2006)
Hum Mutat
, vol.27
, pp. 1174-1184
-
-
Sobacchi, C.1
Marrella, V.2
Rucci, F.3
Vezzoni, P.4
Villa, A.5
-
35
-
-
80052495904
-
Correction of murine Rag1 deficiency by self-inactivating lentiviral vector-mediated gene transfer
-
K. Pike-Overzet, M. Rodijk, Y.-Y. Ng, M.R.M. Baert, C. Lagresle-Peyrou, A. Schambach, F. Zhang, R.C. Hoeben, S. Hacein-Bey-Abina, A.C. Lankester, and et al. Correction of murine Rag1 deficiency by self-inactivating lentiviral vector-mediated gene transfer Leukemia 25 2011 1471 1483
-
(2011)
Leukemia
, vol.25
, pp. 1471-1483
-
-
Pike-Overzet, K.1
Rodijk, M.2
Ng, Y.-Y.3
Baert, M.R.M.4
Lagresle-Peyrou, C.5
Schambach, A.6
Zhang, F.7
Hoeben, R.C.8
Hacein-Bey-Abina, S.9
Lankester, A.C.10
-
36
-
-
0036891848
-
Gene therapy of RAG-2-/- mice: Sustained correction of the immunodeficiency
-
F. Yates, M. Malassis-Séris, D. Stockholm, C. Bouneaud, F. Larousserie, P. Noguiez-Hellin, O. Danos, D.B. Kohn, A. Fischer, J.-P. de Villartay, and et al. Gene therapy of RAG-2-/- mice: sustained correction of the immunodeficiency Blood 100 2002 3942 3949
-
(2002)
Blood
, vol.100
, pp. 3942-3949
-
-
Yates, F.1
Malassis-Séris, M.2
Stockholm, D.3
Bouneaud, C.4
Larousserie, F.5
Noguiez-Hellin, P.6
Danos, O.7
Kohn, D.B.8
Fischer, A.9
De Villartay, J.-P.10
-
37
-
-
84867013112
-
Correction of murine Rag2 severe combined immunodeficiency by lentiviral gene therapy using a codon-optimized RAG2 therapeutic transgene
-
N.P. van Til, H. de Boer, N. Mashamba, A. Wabik, M. Huston, T.P. Visser, E. Fontana, P.L. Poliani, B. Cassani, F. Zhang, and et al. Correction of murine Rag2 severe combined immunodeficiency by lentiviral gene therapy using a codon-optimized RAG2 therapeutic transgene Mol Ther 20 2012 1968 1980
-
(2012)
Mol Ther
, vol.20
, pp. 1968-1980
-
-
Van Til, N.P.1
De Boer, H.2
Mashamba, N.3
Wabik, A.4
Huston, M.5
Visser, T.P.6
Fontana, E.7
Poliani, P.L.8
Cassani, B.9
Zhang, F.10
-
38
-
-
84897389322
-
Recombination-activating gene 1 (Rag1) - Deficient mice with severe combined immunodeficiency treated with lentiviral gene therapy demonstrate autoimmune Omenn-like syndrome
-
N.P. van Til, R. Sarwari, T.P. Visser, and J. Hauer Recombination-activating gene 1 (Rag1) - deficient mice with severe combined immunodeficiency treated with lentiviral gene therapy demonstrate autoimmune Omenn-like syndrome J Allergy Clin Immunol 133 2014 1116 1123
-
(2014)
J Allergy Clin Immunol
, vol.133
, pp. 1116-1123
-
-
Van Til, N.P.1
Sarwari, R.2
Visser, T.P.3
Hauer, J.4
-
39
-
-
84886727845
-
Hemophagocytic lymphohistiocytosis: Advances in pathophysiology, diagnosis, and treatment
-
S. Chandrakasan, and A.H. Filipovich Hemophagocytic lymphohistiocytosis: advances in pathophysiology, diagnosis, and treatment J Pediatr 163 2013 1253 1259
-
(2013)
J Pediatr
, vol.163
, pp. 1253-1259
-
-
Chandrakasan, S.1
Filipovich, A.H.2
-
40
-
-
84924810410
-
Gene therapy for haemophagocytic lymphohistiocytosis
-
C. Booth, M. Carmo, and H. Bobby Gaspar Gene therapy for haemophagocytic lymphohistiocytosis Curr Gene Ther 14 2014 437 446
-
(2014)
Curr Gene Ther
, vol.14
, pp. 437-446
-
-
Booth, C.1
Carmo, M.2
Bobby Gaspar, H.3
-
41
-
-
84926979642
-
Perforin gene transfer into hematopoietic stem cells improves immune dysregulation in murine models of perforin deficiency
-
M. Carmo, K.A. Risma, P. Arumugam, S. Tiwari, A.E. Hontz, C.A. Montiel-Equihua, M.E. Alonso-Ferrero, M.P. Blundell, A. Schambach, C. Baum, and et al. Perforin gene transfer into hematopoietic stem cells improves immune dysregulation in murine models of perforin deficiency Mol Ther 23 2015 737 745
-
(2015)
Mol Ther
, vol.23
, pp. 737-745
-
-
Carmo, M.1
Risma, K.A.2
Arumugam, P.3
Tiwari, S.4
Hontz, A.E.5
Montiel-Equihua, C.A.6
Alonso-Ferrero, M.E.7
Blundell, M.P.8
Schambach, A.9
Baum, C.10
-
42
-
-
78650984696
-
X-linked lymphoproliferative disease due to SAP/SH2D1A deficiency: A multicenter study on the manifestations, management and outcome of the disease
-
C. Booth, K.C. Gilmour, P. Veys, A.R. Gennery, M.A. Slatter, H. Chapel, P.T. Heath, C.G. Steward, O. Smith, A. O'Meara, and et al. X-linked lymphoproliferative disease due to SAP/SH2D1A deficiency: a multicenter study on the manifestations, management and outcome of the disease Blood 117 2011 53 62
-
(2011)
Blood
, vol.117
, pp. 53-62
-
-
Booth, C.1
Gilmour, K.C.2
Veys, P.3
Gennery, A.R.4
Slatter, M.A.5
Chapel, H.6
Heath, P.T.7
Steward, C.G.8
Smith, O.9
O'Meara, A.10
-
43
-
-
84874078294
-
SAP gene transfer restores cellular and humoral immune function in a murine model of X-linked lymphoproliferative disease
-
SAP gene transfer restores cellular and humoral immune function in a murine model of X-linked lymphoproliferative disease Blood 121 2013 1066 1067
-
(2013)
Blood
, vol.121
, pp. 1066-1067
-
-
-
44
-
-
84878668046
-
Advances in stem cell transplantation and gene therapy in the β-hemoglobinopathies
-
E. Payen, and P. Leboulch Advances in stem cell transplantation and gene therapy in the β-hemoglobinopathies Hematol Am Soc Hematol Educ Program 2012 2012 276 283
-
(2012)
Hematol Am Soc Hematol Educ Program
, vol.2012
, pp. 276-283
-
-
Payen, E.1
Leboulch, P.2
-
45
-
-
70449127230
-
Insertional transformation of hematopoietic cells by self-inactivating lentiviral and gammaretroviral vectors
-
U. Modlich, S. Navarro, D. Zychlinski, T. Maetzig, S. Knoess, M.H. Brugman, A. Schambach, S. Charrier, A. Galy, A.J. Thrasher, and et al. 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
Schambach, A.7
Charrier, S.8
Galy, A.9
Thrasher, A.J.10
-
46
-
-
84895427883
-
Preclinical demonstration of lentiviral vector-mediated correction of immunological and metabolic abnormalities in models of adenosine deaminase deficiency
-
D.A. Carbonaro, L. Zhang, X. Jin, C. Montiel-Equihua, S. Geiger, M. Carmo, A. Cooper, L. Fairbanks, M.L. Kaufman, N.I.J. Sebire, and et al. Preclinical demonstration of lentiviral vector-mediated correction of immunological and metabolic abnormalities in models of adenosine deaminase deficiency Mol Ther 22 2014 607 622
-
(2014)
Mol Ther
, vol.22
, pp. 607-622
-
-
Carbonaro, D.A.1
Zhang, L.2
Jin, X.3
Montiel-Equihua, C.4
Geiger, S.5
Carmo, M.6
Cooper, A.7
Fairbanks, L.8
Kaufman, M.L.9
Sebire, N.I.J.10
-
47
-
-
84860718270
-
Decade-long safety and function of retroviral-modified chimeric antigen receptor T cells
-
J. Scholler, T.L. Brady, G. Binder-Scholl, W.-T. Hwang, G. Plesa, K.M. Hege, A.N. Vogel, M. Kalos, J.L. Riley, S.G. Deeks, and et al. Decade-long safety and function of retroviral-modified chimeric antigen receptor T cells Sci Transl Med 4 2012 132ra53
-
(2012)
Sci Transl Med
, vol.4
, pp. 132ra53
-
-
Scholler, J.1
Brady, T.L.2
Binder-Scholl, G.3
Hwang, W.-T.4
Plesa, G.5
Hege, K.M.6
Vogel, A.N.7
Kalos, M.8
Riley, J.L.9
Deeks, S.G.10
-
48
-
-
55249087037
-
Resistance of mature T cells to oncogene transformation
-
S. Newrzela, K. Cornils, Z. Li, C. Baum, M.H. Brugman, M. Hartmann, J. Meyer, S. Hartmann, M.-L. Hansmann, B. Fehse, and et al. Resistance of mature T cells to oncogene transformation Blood 112 2008 2278 2286
-
(2008)
Blood
, vol.112
, pp. 2278-2286
-
-
Newrzela, S.1
Cornils, K.2
Li, Z.3
Baum, C.4
Brugman, M.H.5
Hartmann, M.6
Meyer, J.7
Hartmann, S.8
Hansmann, M.-L.9
Fehse, B.10
-
49
-
-
34948875308
-
Immune dysregulation, polyendocrinopathy, enteropathy, X-linked: Forkhead box protein 3 mutations and lack of regulatory T cells
-
T.R. Torgerson, and H.D. Ochs Immune dysregulation, polyendocrinopathy, enteropathy, X-linked: forkhead box protein 3 mutations and lack of regulatory T cells J Allergy Clin Immunol 120 2007 744 750
-
(2007)
J Allergy Clin Immunol
, vol.120
, pp. 744-750
-
-
Torgerson, T.R.1
Ochs, H.D.2
-
50
-
-
84891673531
-
CD4+ T cells from IPEX patients convert into functional and stable regulatory T cells by FOXP3 gene transfer
-
L. Passerini, E. Rossi Mel, C. Sartirana, G. Fousteri, A. Bondanza, L. Naldini, M.-G. Roncarolo, and R. Bacchetta CD4+ T cells from IPEX patients convert into functional and stable regulatory T cells by FOXP3 gene transfer Sci Transl Med 5 2013 215ra174
-
(2013)
Sci Transl Med
, vol.5
, pp. 215ra174
-
-
Passerini, L.1
Rossi Mel, E.2
Sartirana, C.3
Fousteri, G.4
Bondanza, A.5
Naldini, L.6
Roncarolo, M.-G.7
Bacchetta, R.8
-
51
-
-
0030843493
-
HSV-TK gene transfer into donor lymphocytes for control of allogeneic graft-versus-leukemia
-
C. Bonini, G. Ferrari, S. Verzeletti, P. Servida, E. Zappone, L. Ruggieri, M. Ponzoni, S. Rossini, F. Mavilio, C. Traversari, and et al. HSV-TK gene transfer into donor lymphocytes for control of allogeneic graft-versus-leukemia Science 276 1997 1719 1724
-
(1997)
Science
, vol.276
, pp. 1719-1724
-
-
Bonini, C.1
Ferrari, G.2
Verzeletti, S.3
Servida, P.4
Zappone, E.5
Ruggieri, L.6
Ponzoni, M.7
Rossini, S.8
Mavilio, F.9
Traversari, C.10
-
52
-
-
65349157330
-
Infusion of suicide-gene-engineered donor lymphocytes after family haploidentical haemopoietic stem-cell transplantation for leukaemia (the TK007 trial): A non-randomised phase I-II study
-
F. Ciceri, C. Bonini, M.T.L. Stanghellini, A. Bondanza, C. Traversari, M. Salomoni, L. Turchetto, S. Colombi, M. Bernardi, J. Peccatori, and et al. Infusion of suicide-gene-engineered donor lymphocytes after family haploidentical haemopoietic stem-cell transplantation for leukaemia (the TK007 trial): a non-randomised phase I-II study Lancet Oncol 10 2009 489 500
-
(2009)
Lancet Oncol
, vol.10
, pp. 489-500
-
-
Ciceri, F.1
Bonini, C.2
Stanghellini, M.T.L.3
Bondanza, A.4
Traversari, C.5
Salomoni, M.6
Turchetto, L.7
Colombi, S.8
Bernardi, M.9
Peccatori, J.10
-
53
-
-
84886913160
-
Production and first-in-man use of T cells engineered to express a HSVTK-CD34 sort-suicide gene
-
H. Zhan, K. Gilmour, L. Chan, F. Farzaneh, A.-M. McNicol, J.-H. Xu, S. Adams, B. Fehse, P. Veys, A. Thrasher, and et al. Production and first-in-man use of T cells engineered to express a HSVTK-CD34 sort-suicide gene PLoS ONE 8 2013 e77106
-
(2013)
PLoS ONE
, vol.8
, pp. e77106
-
-
Zhan, H.1
Gilmour, K.2
Chan, L.3
Farzaneh, F.4
McNicol, A.-M.5
Xu, J.-H.6
Adams, S.7
Fehse, B.8
Veys, P.9
Thrasher, A.10
-
54
-
-
84868209465
-
Use of TK-cells in haploidentical hematopoietic stem cell transplantation
-
G. Oliveira, R. Greco, M.T. Lupo-Stanghellini, L. Vago, and C. Bonini Use of TK-cells in haploidentical hematopoietic stem cell transplantation Curr Opin Hematol 19 2012 427 433
-
(2012)
Curr Opin Hematol
, vol.19
, pp. 427-433
-
-
Oliveira, G.1
Greco, R.2
Lupo-Stanghellini, M.T.3
Vago, L.4
Bonini, C.5
-
55
-
-
80455162464
-
Inducible apoptosis as a safety switch for adoptive cell therapy
-
A. Di Stasi, S.-K. Tey, G. Dotti, Y. Fujita, A. Kennedy-Nasser, C. Martinez, K. Straathof, E. Liu, A.G. Durett, B. Grilley, and et al. Inducible apoptosis as a safety switch for adoptive cell therapy N Engl J Med 365 2011 1673 1683
-
(2011)
N Engl J Med
, vol.365
, pp. 1673-1683
-
-
Di Stasi, A.1
Tey, S.-K.2
Dotti, G.3
Fujita, Y.4
Kennedy-Nasser, A.5
Martinez, C.6
Straathof, K.7
Liu, E.8
Durett, A.G.9
Grilley, B.10
-
56
-
-
84933529009
-
Inducible caspase-9 suicide gene controls adverse effects from alloreplete T cells after haploidentical stem cell transplantation
-
X. Zhou, G. Dotti, R.A. Krance, C.A. Martinez, S. Naik, R.T. Kamble, A.G. Durett, O. Dakhova, B. Savoldo, A. Di Stasi, and et al. Inducible caspase-9 suicide gene controls adverse effects from alloreplete T cells after haploidentical stem cell transplantation Blood 125 2015 4103 4113
-
(2015)
Blood
, vol.125
, pp. 4103-4113
-
-
Zhou, X.1
Dotti, G.2
Krance, R.A.3
Martinez, C.A.4
Naik, S.5
Kamble, R.T.6
Durett, A.G.7
Dakhova, O.8
Savoldo, B.9
Di Stasi, A.10
-
57
-
-
13044309472
-
Stable transduction of quiescent CD34(+)CD38(-) human hematopoietic cells by HIV-1-based lentiviral vectors
-
S.S. Case, M.A. Price, C.T. Jordan, X.J. Yu, L. Wang, G. Bauer, D.L. Haas, D. Xu, R. Stripecke, L. Naldini, and et al. Stable transduction of quiescent CD34(+)CD38(-) human hematopoietic cells by HIV-1-based lentiviral vectors Proc Natl Acad Sci U S A 96 1999 2988 2993
-
(1999)
Proc Natl Acad Sci U S A
, vol.96
, pp. 2988-2993
-
-
Case, S.S.1
Price, M.A.2
Jordan, C.T.3
Yu, X.J.4
Wang, L.5
Bauer, G.6
Haas, D.L.7
Xu, D.8
Stripecke, R.9
Naldini, L.10
-
58
-
-
0033034215
-
Thrombopoietin, flt3, and kit ligands together suppress apoptosis of human mobilized CD34+ cells and recruit primitive CD34+ Thy-1+ cells into rapid division
-
L.J. Murray, J.C. Young, L.J. Osborne, K.M. Luens, R. Scollay, and B.L. Hill Thrombopoietin, flt3, and kit ligands together suppress apoptosis of human mobilized CD34+ cells and recruit primitive CD34+ Thy-1+ cells into rapid division Exp Hematol 27 1999 1019 1028
-
(1999)
Exp Hematol
, vol.27
, pp. 1019-1028
-
-
Murray, L.J.1
Young, J.C.2
Osborne, L.J.3
Luens, K.M.4
Scollay, R.5
Hill, B.L.6
-
59
-
-
84876926824
-
LDL receptor and its family members serve as the cellular receptors for vesicular stomatitis virus
-
D. Finkelshtein, A. Werman, D. Novick, S. Barak, and M. Rubinstein LDL receptor and its family members serve as the cellular receptors for vesicular stomatitis virus Proc Natl Acad Sci U S A 110 2013 7306 7311
-
(2013)
Proc Natl Acad Sci U S A
, vol.110
, pp. 7306-7311
-
-
Finkelshtein, D.1
Werman, A.2
Novick, D.3
Barak, S.4
Rubinstein, M.5
-
60
-
-
0036493384
-
Homing efficiency, cell cycle kinetics, and survival of quiescent and cycling human CD34(+) cells transplanted into conditioned NOD/SCID recipients
-
A. Jetmore, P.A. Plett, X. Tong, F.M. Wolber, R. Breese, R. Abonour, C.M. Orschell-Traycoff, and E.F. Srour Homing efficiency, cell cycle kinetics, and survival of quiescent and cycling human CD34(+) cells transplanted into conditioned NOD/SCID recipients Blood 99 2002 1585 1593
-
(2002)
Blood
, vol.99
, pp. 1585-1593
-
-
Jetmore, A.1
Plett, P.A.2
Tong, X.3
Wolber, F.M.4
Breese, R.5
Abonour, R.6
Orschell-Traycoff, C.M.7
Srour, E.F.8
-
61
-
-
1542373663
-
Impaired bone marrow homing of cytokine-activated CD34+ cells in the NOD/SCID model
-
F. Ahmed, S.J. Ings, A.R. Pizzey, M.P. Blundell, A.J. Thrasher, H.T. Ye, A. Fahey, D.C. Linch, and K.L. Yong Impaired bone marrow homing of cytokine-activated CD34+ cells in the NOD/SCID model Blood 103 2004 2079 2087
-
(2004)
Blood
, vol.103
, pp. 2079-2087
-
-
Ahmed, F.1
Ings, S.J.2
Pizzey, A.R.3
Blundell, M.P.4
Thrasher, A.J.5
Ye, H.T.6
Fahey, A.7
Linch, D.C.8
Yong, K.L.9
-
62
-
-
84907303748
-
Baboon envelope pseudotyped LVs outperform VSV-G-LVs for gene transfer into early-cytokine-stimulated and resting HSCs
-
A. Girard-Gagnepain, F. Amirache, C. Costa, C. Lévy, C. Frecha, F. Fusil, D. Nègre, D. Lavillette, F.-L. Cosset, and E. Verhoeyen Baboon envelope pseudotyped LVs outperform VSV-G-LVs for gene transfer into early-cytokine-stimulated and resting HSCs Blood 124 2014 1221 1231
-
(2014)
Blood
, vol.124
, pp. 1221-1231
-
-
Girard-Gagnepain, A.1
Amirache, F.2
Costa, C.3
Lévy, C.4
Frecha, C.5
Fusil, F.6
Nègre, D.7
Lavillette, D.8
Cosset, F.-L.9
Verhoeyen, E.10
-
63
-
-
84899683375
-
Mystery solved: VSV-G-LVs do not allow efficient gene transfer into unstimulated T cells, B cells, and HSCs because they lack the LDL receptor
-
F. Amirache, C. Lévy, C. Costa, P.-E. Mangeot, B.E. Torbett, C.X. Wang, D. Nègre, F.-L. Cosset, and E. Verhoeyen Mystery solved: VSV-G-LVs do not allow efficient gene transfer into unstimulated T cells, B cells, and HSCs because they lack the LDL receptor Blood 123 2014 1422 1424
-
(2014)
Blood
, vol.123
, pp. 1422-1424
-
-
Amirache, F.1
Lévy, C.2
Costa, C.3
Mangeot, P.-E.4
Torbett, B.E.5
Wang, C.X.6
Nègre, D.7
Cosset, F.-L.8
Verhoeyen, E.9
-
64
-
-
84920553698
-
CD133-targeted gene transfer into long-term repopulating hematopoietic stem cells
-
C. Brendel, B. Goebel, A. Daniela, M. Brugman, S. Kneissl, J. Schwäble, K.B. Kaufmann, U. Müller-Kuller, H. Kunkel, L. Chen-Wichmann, and et al. CD133-targeted gene transfer into long-term repopulating hematopoietic stem cells Mol Ther 23 2015 63 70
-
(2015)
Mol Ther
, vol.23
, pp. 63-70
-
-
Brendel, C.1
Goebel, B.2
Daniela, A.3
Brugman, M.4
Kneissl, S.5
Schwäble, J.6
Kaufmann, K.B.7
Müller-Kuller, U.8
Kunkel, H.9
Chen-Wichmann, L.10
-
65
-
-
84924346983
-
CD105 is a surface marker for receptor-targeted gene transfer into human long-term repopulating hematopoietic stem cells
-
S.-K. Kays, K.B. Kaufmann, T. Abel, C. Brendel, H. Bonig, M. Grez, C.J. Buchholz, and S. Kneissl CD105 is a surface marker for receptor-targeted gene transfer into human long-term repopulating hematopoietic stem cells Stem Cells Dev 24 2015 714 723
-
(2015)
Stem Cells Dev
, vol.24
, pp. 714-723
-
-
Kays, S.-K.1
Kaufmann, K.B.2
Abel, T.3
Brendel, C.4
Bonig, H.5
Grez, M.6
Buchholz, C.J.7
Kneissl, S.8
-
66
-
-
78049345098
-
Specific gene transfer to neurons, endothelial cells and hematopoietic progenitors with lentiviral vectors
-
B. Anliker, T. Abel, S. Kneissl, J. Hlavaty, A. Caputi, J. Brynza, I.C. Schneider, R.C. Münch, H. Petznek, R.E. Kontermann, and et al. Specific gene transfer to neurons, endothelial cells and hematopoietic progenitors with lentiviral vectors Nat Methods 7 2010 929 935
-
(2010)
Nat Methods
, vol.7
, pp. 929-935
-
-
Anliker, B.1
Abel, T.2
Kneissl, S.3
Hlavaty, J.4
Caputi, A.5
Brynza, J.6
Schneider, I.C.7
Münch, R.C.8
Petznek, H.9
Kontermann, R.E.10
-
67
-
-
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
-
Y.D. Korin, and J.A. Zack 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
-
68
-
-
0034690616
-
Retrovirus-mediated gene transfer in primary T lymphocytes: Influence of the transduction/selection process and of ex vivo expansion on the T cell receptor beta chain hypervariable region repertoire
-
C. Ferrand, E. Robinet, E. Contassot, J.M. Certoux, A. Lim, P. Hervé, and P. Tiberghien Retrovirus-mediated gene transfer in primary T lymphocytes: influence of the transduction/selection process and of ex vivo expansion on the T cell receptor beta chain hypervariable region repertoire Hum Gene Ther 11 2000 1151 1164
-
(2000)
Hum Gene Ther
, vol.11
, pp. 1151-1164
-
-
Ferrand, C.1
Robinet, E.2
Contassot, E.3
Certoux, J.M.4
Lim, A.5
Hervé, P.6
Tiberghien, P.7
-
69
-
-
0037441766
-
Immunologic potential of donor lymphocytes expressing a suicide gene for early immune reconstitution after hematopoietic T-cell-depleted stem cell transplantation
-
S. Marktel, Z. Magnani, F. Ciceri, S. Cazzaniga, S.R. Riddell, C. Traversari, C. Bordignon, and C. Bonini Immunologic potential of donor lymphocytes expressing a suicide gene for early immune reconstitution after hematopoietic T-cell-depleted stem cell transplantation Blood 101 2003 1290 1298
-
(2003)
Blood
, vol.101
, pp. 1290-1298
-
-
Marktel, S.1
Magnani, Z.2
Ciceri, F.3
Cazzaniga, S.4
Riddell, S.R.5
Traversari, C.6
Bordignon, C.7
Bonini, C.8
-
70
-
-
0037443424
-
IL-7 surface-engineered lentiviral vectors promote survival and efficient gene transfer in resting primary T lymphocytes
-
E. Verhoeyen, V. Dardalhon, O. Ducrey-Rundquist, D. Trono, N. Taylor, and F.-L. Cosset IL-7 surface-engineered lentiviral vectors promote survival and efficient gene transfer in resting primary T lymphocytes Blood 101 2003 2167 2174
-
(2003)
Blood
, vol.101
, pp. 2167-2174
-
-
Verhoeyen, E.1
Dardalhon, V.2
Ducrey-Rundquist, O.3
Trono, D.4
Taylor, N.5
Cosset, F.-L.6
-
71
-
-
58149380748
-
Stable transduction of quiescent T cells without induction of cycle progression by a novel lentiviral vector pseudotyped with measles virus glycoproteins
-
C. Frecha, C. Costa, D. Nègre, E. Gauthier, S.J. Russell, F.-L. Cosset, and E. Verhoeyen Stable transduction of quiescent T cells without induction of cycle progression by a novel lentiviral vector pseudotyped with measles virus glycoproteins Blood 112 2008 4843 4852
-
(2008)
Blood
, vol.112
, pp. 4843-4852
-
-
Frecha, C.1
Costa, C.2
Nègre, D.3
Gauthier, E.4
Russell, S.J.5
Cosset, F.-L.6
Verhoeyen, E.7
-
72
-
-
79958119978
-
Measles virus glycoprotein-pseudotyped lentiviral vector-mediated gene transfer into quiescent lymphocytes requires binding to both SLAM and CD46 entry receptors
-
C. Frecha, C. Lévy, C. Costa, D. Nègre, F. Amirache, R. Buckland, S.J. Russell, F.-L. Cosset, and E. Verhoeyen Measles virus glycoprotein-pseudotyped lentiviral vector-mediated gene transfer into quiescent lymphocytes requires binding to both SLAM and CD46 entry receptors J Virol 85 2011 5975 5985
-
(2011)
J Virol
, vol.85
, pp. 5975-5985
-
-
Frecha, C.1
Lévy, C.2
Costa, C.3
Nègre, D.4
Amirache, F.5
Buckland, R.6
Russell, S.J.7
Cosset, F.-L.8
Verhoeyen, E.9
-
73
-
-
84878746105
-
Efficient lentiviral transduction and transgene expression in primary human B cells
-
U. Mock, R. Thiele, A. Uhde, B. Fehse, and S. Horn Efficient lentiviral transduction and transgene expression in primary human B cells Hum Gene Ther Methods 23 2012 408 415
-
(2012)
Hum Gene Ther Methods
, vol.23
, pp. 408-415
-
-
Mock, U.1
Thiele, R.2
Uhde, A.3
Fehse, B.4
Horn, S.5
-
74
-
-
84867743649
-
Development of nuclease-mediated site-specific genome modification
-
S.E. Wirt, and M.H. Porteus Development of nuclease-mediated site-specific genome modification Curr Opin Immunol 24 2012 609 616
-
(2012)
Curr Opin Immunol
, vol.24
, pp. 609-616
-
-
Wirt, S.E.1
Porteus, M.H.2
-
75
-
-
77955867185
-
Genome editing with engineered zinc finger nucleases
-
F.D. Urnov, E.J. Rebar, M.C. Holmes, H.S. Zhang, and P.D. Gregory Genome editing with engineered zinc finger nucleases Nat Rev Genet 11 2010 636 646
-
(2010)
Nat Rev Genet
, vol.11
, pp. 636-646
-
-
Urnov, F.D.1
Rebar, E.J.2
Holmes, M.C.3
Zhang, H.S.4
Gregory, P.D.5
-
76
-
-
80053343092
-
TAL effectors: Customizable proteins for DNA targeting
-
A.J. Bogdanove, and D.F. Voytas TAL effectors: customizable proteins for DNA targeting Science 333 2011 1843 1846
-
(2011)
Science
, vol.333
, pp. 1843-1846
-
-
Bogdanove, A.J.1
Voytas, D.F.2
-
77
-
-
79951694132
-
Meganucleases and other tools for targeted genome engineering: Perspectives and challenges for gene therapy
-
G. Silva, L. Poirot, R. Galetto, J. Smith, G. Montoya, P. Duchateau, and F. Pâques Meganucleases and other tools for targeted genome engineering: perspectives and challenges for gene therapy Curr Gene Ther 11 2011 11 27
-
(2011)
Curr Gene Ther
, vol.11
, pp. 11-27
-
-
Silva, G.1
Poirot, L.2
Galetto, R.3
Smith, J.4
Montoya, G.5
Duchateau, P.6
Pâques, F.7
-
78
-
-
84865070369
-
A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity
-
M. Jinek, K. Chylinski, I. Fonfara, M. Hauer, J.A. Doudna, and E. Charpentier A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity Science 337 2012 816 821
-
(2012)
Science
, vol.337
, pp. 816-821
-
-
Jinek, M.1
Chylinski, K.2
Fonfara, I.3
Hauer, M.4
Doudna, J.A.5
Charpentier, E.6
-
79
-
-
84873734105
-
RNA-guided human genome engineering via Cas9
-
P. Mali, L. Yang, K.M. Esvelt, J. Aach, M. Guell, J.E. DiCarlo, J.E. Norville, and G.M. Church RNA-guided human genome engineering via Cas9 Science 339 2013 823 826
-
(2013)
Science
, vol.339
, pp. 823-826
-
-
Mali, P.1
Yang, L.2
Esvelt, K.M.3
Aach, J.4
Guell, M.5
DiCarlo, J.E.6
Norville, J.E.7
Church, G.M.8
-
80
-
-
84873729095
-
Multiplex genome engineering using CRISPR/Cas systems
-
L. Cong, F.A. Ran, D. Cox, S. Lin, R. Barretto, N. Habib, P.D. Hsu, X. Wu, W. Jiang, L.A. Marraffini, and et al. Multiplex genome engineering using CRISPR/Cas systems Science 339 2013 819 823
-
(2013)
Science
, vol.339
, pp. 819-823
-
-
Cong, L.1
Ran, F.A.2
Cox, D.3
Lin, S.4
Barretto, R.5
Habib, N.6
Hsu, P.D.7
Wu, X.8
Jiang, W.9
Marraffini, L.A.10
-
81
-
-
84913594397
-
Genome editing. The new frontier of genome engineering with CRISPR-Cas9
-
J.A. Doudna, and E. Charpentier Genome editing. The new frontier of genome engineering with CRISPR-Cas9 Science 346 2014 1258096
-
(2014)
Science
, vol.346
, pp. 1258096
-
-
Doudna, J.A.1
Charpentier, E.2
-
82
-
-
84895487305
-
Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV
-
P. Tebas, D. Stein, W.W. Tang, I. Frank, S.Q. Wang, G. Lee, S.K. Spratt, R.T. Surosky, M.A. Giedlin, G. Nichol, and et al. Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV N Engl J Med 370 2014 901 910
-
(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
Spratt, S.K.7
Surosky, R.T.8
Giedlin, M.A.9
Nichol, G.10
-
83
-
-
35948946526
-
Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery
-
A. Lombardo, P. Genovese, C.M. Beausejour, S. Colleoni, Y.-L. Lee, K.A. Kim, D. Ando, F.D. Urnov, C. Galli, P.D. Gregory, and et al. Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery Nat Biotechnol 25 2007 1298 1306
-
(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
Ando, D.7
Urnov, F.D.8
Galli, C.9
Gregory, P.D.10
-
84
-
-
84902315464
-
Targeted genome editing in human repopulating haematopoietic stem cells
-
P. Genovese, G. Schiroli, G. Escobar, T. Di Tomaso, C. Firrito, A. Calabria, D. Moi, R. Mazzieri, C. Bonini, M.C. Holmes, and et al. Targeted genome editing in human repopulating haematopoietic stem cells Nature 510 2014 235 240
-
(2014)
Nature
, vol.510
, pp. 235-240
-
-
Genovese, P.1
Schiroli, G.2
Escobar, G.3
Di Tomaso, T.4
Firrito, C.5
Calabria, A.6
Moi, D.7
Mazzieri, R.8
Bonini, C.9
Holmes, M.C.10
-
85
-
-
85044694762
-
Targeted genome editing by lentiviral protein transduction of zinc-finger and TAL-effector nucleases
-
Y. Cai, R.O. Bak, and J.G. Mikkelsen Targeted genome editing by lentiviral protein transduction of zinc-finger and TAL-effector nucleases Elife 3 2014 e01911
-
(2014)
Elife
, vol.3
, pp. e01911
-
-
Cai, Y.1
Bak, R.O.2
Mikkelsen, J.G.3
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