-
1
-
-
0035832510
-
Primary immunodeficiency diseases: An experimental model for molecular medicine
-
Fischer A. Primary immunodeficiency diseases: an experimental model for molecular medicine. Lancet 2001;357:1863-9.
-
(2001)
Lancet
, vol.357
, pp. 1863-1869
-
-
Fischer, A.1
-
2
-
-
0037442176
-
Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies: Report of the European experience 1968-99
-
Antoine C, Muller S, Cant A, et al. Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies: report of the European experience 1968-99. Lancet 2003;361:553-60.
-
(2003)
Lancet
, vol.361
, pp. 553-560
-
-
Antoine, C.1
Muller, S.2
Cant, A.3
-
3
-
-
0033580206
-
Hematopoietic stem-cell transplantation for the treatment of severe combined immunodeficiency
-
Buckley RH, Schiff SE, Schiff RI, et al. Hematopoietic stem-cell transplantation for the treatment of severe combined immunodeficiency. N Engl J Med 1999;340:508-16.
-
(1999)
N Engl J Med
, vol.340
, pp. 508-516
-
-
Buckley, R.H.1
Schiff, S.E.2
Schiff, R.I.3
-
4
-
-
0029902033
-
Spontaneous in vivo reversion to normal of an inherited mutation in a patient with adenosine deaminase deficiency
-
Hirschhorn R, Yang DR, Puck JM, et al. Spontaneous in vivo reversion to normal of an inherited mutation in a patient with adenosine deaminase deficiency. Nat Genet 1996;13:290-5.
-
(1996)
Nat Genet
, vol.13
, pp. 290-295
-
-
Hirschhorn, R.1
Yang, D.R.2
Puck, J.M.3
-
5
-
-
12944284582
-
Diversity, functionality, and stability of the T cell repertoire derived in vivo from a single human T cell precursor
-
Bousso P, Wahn V, Douagi I, et al. Diversity, functionality, and stability of the T cell repertoire derived in vivo from a single human T cell precursor. Proc Natl Acad Sci U S A 2000;97:274-8.
-
(2000)
Proc Natl Acad Sci U S A
, vol.97
, pp. 274-278
-
-
Bousso, P.1
Wahn, V.2
Douagi, I.3
-
6
-
-
0037162715
-
HIV-1 integration in the human genome favors active genes and local hotspots
-
Schroder AR, Shinn P, Chen H, et al. HIV-1 integration in the human genome favors active genes and local hotspots. Cell 2002;110:521-9.
-
(2002)
Cell
, vol.110
, pp. 521-529
-
-
Schroder, A.R.1
Shinn, P.2
Chen, H.3
-
7
-
-
0037841763
-
Transcription start regions in the human genome are favored targets for MLV integration
-
Wu X, Li Y, Crise B, et al. Transcription start regions in the human genome are favored targets for MLV integration. Science 2003;300:1749-51.
-
(2003)
Science
, vol.300
, pp. 1749-1751
-
-
Wu, X.1
Li, Y.2
Crise, B.3
-
8
-
-
0034724857
-
Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease
-
Cavazzana-Calvo M, Hacein-Bey S, de Saint BG, et al. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 2000;288:669-72.
-
(2000)
Science
, vol.288
, pp. 669-672
-
-
Cavazzana-Calvo, M.1
Hacein-Bey, S.2
de Saint, B.G.3
-
9
-
-
19944421384
-
Gene therapy of X-linlced severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector
-
Gaspar HB, Parsley KL, Howe S, et al. Gene therapy of X-linlced severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector. Lancet 2004;364:2181-7.
-
(2004)
Lancet
, vol.364
, pp. 2181-2187
-
-
Gaspar, H.B.1
Parsley, K.L.2
Howe, S.3
-
10
-
-
0037129435
-
Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy
-
Hacein-Bey-Abina S, Le Deist F, Carlier F, et al. Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. N Engl J Med 2002;346:1185-93.
-
(2002)
N Engl J Med
, vol.346
, pp. 1185-1193
-
-
Hacein-Bey-Abina, S.1
Le Deist, F.2
Carlier, F.3
-
12
-
-
21144439712
-
Failure of SCID-X1 gene therapy in older patients
-
Thrasher AJ, Hocein-Bey-Abina S, Caspar HB, et al. Failure of SCID-X1 gene therapy in older patients. Blood 2005;105:4255-7.
-
(2005)
Blood
, vol.105
, pp. 4255-4257
-
-
Thrasher, A.J.1
Hocein-Bey-Abina, S.2
Caspar, H.B.3
-
13
-
-
0028807727
-
T lymphocyte-directed gene therapy for ADA-SCID: Initial trial results after 4 years
-
Bloese RM, Culver KW, Miller AD, et al. T lymphocyte-directed gene therapy for ADA-SCID: initial trial results after 4 years. Science 1995;270:475-80.
-
(1995)
Science
, vol.270
, pp. 475-480
-
-
Bloese, R.M.1
Culver, K.W.2
Miller, A.D.3
-
14
-
-
0038446699
-
Persistence and expression of the adenosine deaminase gene for 12 years and immune reaction to gene transfer components: Long-term results of the first clinical gene therapy trial
-
Muul LM, Tuschong LM, Soenen SL, et al. Persistence and expression of the adenosine deaminase gene for 12 years and immune reaction to gene transfer components: long-term results of the first clinical gene therapy trial Blood 2003;101:2563-9.
-
(2003)
Blood
, vol.101
, pp. 2563-2569
-
-
Muul, L.M.1
Tuschong, L.M.2
Soenen, S.L.3
-
15
-
-
0028789792
-
Gene therapy in peripheral blood lymphocytes and bone marrow for ADA-immunodeficient patients
-
Bordignon C, Notarangelo LD, Nobili N, et al. Gene therapy in peripheral blood lymphocytes and bone marrow for ADA-immunodeficient patients. Science 1995;270:470-5.
-
(1995)
Science
, vol.270
, pp. 470-475
-
-
Bordignon, C.1
Notarangelo, L.D.2
Nobili, N.3
-
16
-
-
9244243165
-
Bone marrow gene transfer in three patients with adenosine deaminase deficiency
-
Hoogerbrugge PM, van Beusechem VW, Fischer A, et al. Bone marrow gene transfer in three patients with adenosine deaminase deficiency. Gene Ther 1996;3:179-83.
-
(1996)
Gene Ther
, vol.3
, pp. 179-183
-
-
Hoogerbrugge, P.M.1
van Beusechem, V.W.2
Fischer, A.3
-
17
-
-
0031593688
-
T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+ cells in ADA-deficient SCID neonates
-
Kohn DB, Hershfield MS, Carbonaro D, et al. T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+ cells in ADA-deficient SCID neonates. Nat Med 1998;4:775-80.
-
(1998)
Nat Med
, vol.4
, pp. 775-780
-
-
Kohn, D.B.1
Hershfield, M.S.2
Carbonaro, D.3
-
18
-
-
18444376150
-
Immune reconstitution in ADA-SCID after PBL gene therapy and discontinuation of enzyme replacement
-
Aiuti A, Vai S, Mortellaro A, et al. Immune reconstitution in ADA-SCID after PBL gene therapy and discontinuation of enzyme replacement. Nat Med 2002;8:423-5.
-
(2002)
Nat Med
, vol.8
, pp. 423-425
-
-
Aiuti, A.1
Vai, S.2
Mortellaro, A.3
-
19
-
-
33846196566
-
Prolonged pancytopenia in a gene therapy patient with ADA-deficient SCID and trisomy 8 mosaicism - a case report
-
Engel BC, Podsakoff GM, Ireland JL, et al. Prolonged pancytopenia in a gene therapy patient with ADA-deficient SCID and trisomy 8 mosaicism - a case report. Blood 2007;109(2):503-6.
-
(2007)
Blood
, vol.109
, Issue.2
, pp. 503-506
-
-
Engel, B.C.1
Podsakoff, G.M.2
Ireland, J.L.3
-
20
-
-
0037189401
-
Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning
-
Aiufi A, Slavin S, Aker M, et al. Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science 2002;296:2410-13.
-
(2002)
Science
, vol.296
, pp. 2410-2413
-
-
Aiufi, A.1
Slavin, S.2
Aker, M.3
-
21
-
-
33748413936
-
Successful reconstitution of immunity in ADA-SOD by stem cell gene therapy following cessation of PEG-ADA and use of mild preconditioning
-
Caspar HB, Biorkegren E, Parsley K, et al. Successful reconstitution of immunity in ADA-SOD by stem cell gene therapy following cessation of PEG-ADA and use of mild preconditioning. Mol Ther 2006;14:505-13.
-
(2006)
Mol Ther
, vol.14
, pp. 505-513
-
-
Caspar, H.B.1
Biorkegren, E.2
Parsley, K.3
-
22
-
-
0037448352
-
A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency
-
Hocein-Bey-Abina S, von Kalle C, Schmidt M, et al. A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N Engl J Med 2003;348:255-6.
-
(2003)
N Engl J Med
, vol.348
, pp. 255-256
-
-
Hocein-Bey-Abina, S.1
von Kalle, C.2
Schmidt, M.3
-
23
-
-
0142084745
-
LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1
-
Hacein-Bey-Abina S, von Kalle C, Schmidt M, et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 2003;302:415-19.
-
(2003)
Science
, vol.302
, pp. 415-419
-
-
Hacein-Bey-Abina, S.1
von Kalle, C.2
Schmidt, M.3
-
24
-
-
33646361846
-
Gene therapy: Therapeutic gene causing lymphoma
-
Woods NB, Bottero V, Schmidt M, et al. Gene therapy: therapeutic gene causing lymphoma. Nature 2006;440:1123.
-
(2006)
Nature
, vol.440
, pp. 1123
-
-
Woods, N.B.1
Bottero, V.2
Schmidt, M.3
-
25
-
-
33748939587
-
Gene therapy: X-SCID transgene leukaemogenicity
-
Thrasher AJ, Gaspar HB, Baum C, et al. Gene therapy: X-SCID transgene leukaemogenicity. Nature 2006;443:E5-6.
-
(2006)
Nature
, vol.443
-
-
Thrasher, A.J.1
Gaspar, H.B.2
Baum, C.3
-
26
-
-
33750624015
-
Cell-culture assays reveal the importance of retroviral vector design for insertional genotoxicity
-
Modlich U, Bohne J, Schmidt M, et al. Cell-culture assays reveal the importance of retroviral vector design for insertional genotoxicity. Blood 2006;108:2545-53.
-
(2006)
Blood
, vol.108
, pp. 2545-2553
-
-
Modlich, U.1
Bohne, J.2
Schmidt, M.3
-
27
-
-
33645734405
-
Correction of X-linked chronic granulornatous disease by gene therapy, augmented by 'insertional activation of MDS1-EV11, PRDM16 or SETBP1
-
Ott MG, Schmidt M, Schwarzwaelder K, et al. Correction of X-linked chronic granulornatous disease by gene therapy, augmented by 'insertional activation of MDS1-EV11, PRDM16 or SETBP1. Nat Med 2006,12:401-9.
-
(2006)
Nat Med
, vol.12
, pp. 401-409
-
-
Ott, M.G.1
Schmidt, M.2
Schwarzwaelder, K.3
-
28
-
-
33845993292
-
Stem cell collection and gene transfer in Fanconi anemia
-
Kelly PF, Radtke S, Kalle C, et al. Stem cell collection and gene transfer in Fanconi anemia. Mol Ther 2007;15:211-19.
-
(2007)
Mol Ther
, vol.15
, pp. 211-219
-
-
Kelly, P.F.1
Radtke, S.2
Kalle, C.3
-
29
-
-
33751251058
-
Gene transfer in humans using a conditionally replicating lentiviral vector
-
Levine BL, Humeau LM, Boyer J, et al. Gene transfer in humans using a conditionally replicating lentiviral vector. Proc Natl Acad Sci U S A 2006;103:17372-7.
-
(2006)
Proc Natl Acad Sci U S A
, vol.103
, pp. 17372-17377
-
-
Levine, B.L.1
Humeau, L.M.2
Boyer, J.3
-
30
-
-
33749624177
-
Cancer regression in patients after transfer of genetically engineered lymphocytes
-
Morgan RA, Dudley ME, Wunderlich JR, et al. Cancer regression in patients after transfer of genetically engineered lymphocytes. Science 2006;314:126-9.
-
(2006)
Science
, vol.314
, pp. 126-129
-
-
Morgan, R.A.1
Dudley, M.E.2
Wunderlich, J.R.3
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