-
1
-
-
0034724857
-
Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease
-
Cavazzana-Calvo M, Hacein-Bey S, de Saint Basile G, Gross F, Yvon E, et al. (2000) Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 288: 669-672.
-
(2000)
Science
, vol.288
, pp. 669-672
-
-
Cavazzana-Calvo, M.1
Hacein-Bey, S.2
de Saint Basile, G.3
Gross, F.4
Yvon, E.5
-
2
-
-
0037189401
-
Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning
-
Aiuti A, Slavin S, Aker M, Ficara F, Deola S, et al. (2002) Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science 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
-
3
-
-
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, et al. (2004) Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector. Lancet 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
-
4
-
-
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, et al. (2006) Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nat Med 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
-
5
-
-
78149482538
-
Stem-Cell Gene Therapy for the Wiskott-Aldrich Syndrome
-
Boztug K, Schmidt M, Schwarzer A, Banerjee PP, Díez IsA, et al. (2010) Stem-Cell Gene Therapy for the Wiskott-Aldrich Syndrome. New England Journal of Medicine 363: 1918-1927.
-
(2010)
New England Journal of Medicine
, vol.363
, pp. 1918-1927
-
-
Boztug, K.1
Schmidt, M.2
Schwarzer, A.3
Banerjee, P.P.4
Díez, I.S.A.5
-
6
-
-
27944449787
-
Insertional mutagenesis identifies genes that promote the immortalization of primary bone marrow progenitor cells
-
Du Y, Jenkins NA, Copeland NG, (2005) Insertional mutagenesis identifies genes that promote the immortalization of primary bone marrow progenitor cells. Blood 106: 3932-3939.
-
(2005)
Blood
, vol.106
, pp. 3932-3939
-
-
Du, Y.1
Jenkins, N.A.2
Copeland, N.G.3
-
7
-
-
20044396722
-
Distinct genomic integration of MLV and SIV vectors in primate hematopoietic stem and progenitor cells
-
Hematti P, Hong BK, Ferguson C, Adler R, Hanawa H, et al. (2004) Distinct genomic integration of MLV and SIV vectors in primate hematopoietic stem and progenitor cells. PLoS Biol 2: e423.
-
(2004)
PLoS Biol
, vol.2
-
-
Hematti, P.1
Hong, B.K.2
Ferguson, C.3
Adler, R.4
Hanawa, H.5
-
8
-
-
25144490586
-
Recurrent retroviral vector integration at the Mds1/Evi1 locus in nonhuman primate hematopoietic cells
-
Calmels B, Ferguson C, Laukkanen MO, Adler R, Faulhaber M, et al. (2005) Recurrent retroviral vector integration at the Mds1/Evi1 locus in nonhuman primate hematopoietic cells. Blood 106: 2530-2533.
-
(2005)
Blood
, vol.106
, pp. 2530-2533
-
-
Calmels, B.1
Ferguson, C.2
Laukkanen, M.O.3
Adler, R.4
Faulhaber, M.5
-
9
-
-
21044448037
-
Clonal dominance of hematopoietic stem cells triggered by retroviral gene marking
-
Kustikova O, Fehse B, Modlich U, Yang M, Dullmann J, et al. (2005) Clonal dominance of hematopoietic stem cells triggered by retroviral gene marking. Science 308: 1171-1174.
-
(2005)
Science
, vol.308
, pp. 1171-1174
-
-
Kustikova, O.1
Fehse, B.2
Modlich, U.3
Yang, M.4
Dullmann, J.5
-
10
-
-
0037134031
-
Murine leukemia induced by retroviral gene marking
-
Li Z, Dullmann J, Schiedlmeier B, Schmidt M, von Kalle C, et al. (2002) Murine leukemia induced by retroviral gene marking. Science 296: 497.
-
(2002)
Science
, vol.296
, pp. 497
-
-
Li, Z.1
Dullmann, J.2
Schiedlmeier, B.3
Schmidt, M.4
von Kalle, C.5
-
11
-
-
21144437399
-
Leukemias following retroviral transfer of multidrug resistance 1 (MDR1) are driven by combinatorial insertional mutagenesis
-
Modlich U, Kustikova OS, Schmidt M, Rudolph C, Meyer J, et al. (2005) Leukemias following retroviral transfer of multidrug resistance 1 (MDR1) are driven by combinatorial insertional mutagenesis. Blood 105: 4235-4246.
-
(2005)
Blood
, vol.105
, pp. 4235-4246
-
-
Modlich, U.1
Kustikova, O.S.2
Schmidt, M.3
Rudolph, C.4
Meyer, J.5
-
12
-
-
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, et al. (2006) Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration. Nat Biotechnol 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
-
13
-
-
34249906359
-
Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy
-
Deichmann A, Hacein-Bey-Abina S, Schmidt M, Garrigue A, Brugman MH, et al. (2007) Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy. J Clin Invest 117: 2225-2232.
-
(2007)
J Clin Invest
, vol.117
, pp. 2225-2232
-
-
Deichmann, A.1
Hacein-Bey-Abina, S.2
Schmidt, M.3
Garrigue, A.4
Brugman, M.H.5
-
14
-
-
34547651095
-
Gammaretrovirus-mediated correction of SCID-X1 is associated with skewed vector integration site distribution in vivo
-
Schwarzwaelder K, Howe SJ, Schmidt M, Brugman MH, Deichmann A, et al. (2007) Gammaretrovirus-mediated correction of SCID-X1 is associated with skewed vector integration site distribution in vivo. J Clin Invest 117: 2241-2249.
-
(2007)
J Clin Invest
, vol.117
, pp. 2241-2249
-
-
Schwarzwaelder, K.1
Howe, S.J.2
Schmidt, M.3
Brugman, M.H.4
Deichmann, A.5
-
15
-
-
51349158298
-
Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients
-
Howe SJ, Mansour MR, Schwarzwaelder K, Bartholomae C, Hubank M, et al. (2008) Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients. J Clin Invest 118: 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
-
16
-
-
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, et al. (2003) A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N Engl J Med 348: 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
-
17
-
-
76249131912
-
Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease
-
Stein S, Ott MG, Schultze-Strasser S, Jauch A, Burwinkel B, et al. (2010) Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease. Nat Med 16: 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
-
18
-
-
34250185398
-
Advances in the treatment of Chronic Granulomatous Disease by gene therapy
-
Ott MG, Seger R, Stein S, Siler U, Hoelzer D, et al. (2007) Advances in the treatment of Chronic Granulomatous Disease by gene therapy. Curr Gene Ther 7: 155-161.
-
(2007)
Curr Gene Ther
, vol.7
, pp. 155-161
-
-
Ott, M.G.1
Seger, R.2
Stein, S.3
Siler, U.4
Hoelzer, D.5
-
19
-
-
71549135258
-
Comprehensive genomic access to vector integration in clinical gene therapy
-
Gabriel R, Eckenberg R, Paruzynski A, Bartholomae CC, Nowrouzi A, et al. (2009) Comprehensive genomic access to vector integration in clinical gene therapy. Nat Med 15: 1431-1436.
-
(2009)
Nat Med
, vol.15
, pp. 1431-1436
-
-
Gabriel, R.1
Eckenberg, R.2
Paruzynski, A.3
Bartholomae, C.C.4
Nowrouzi, A.5
-
20
-
-
77955220225
-
Genome-wide high-throughput integrome analyses by nrLAM-PCR and next-generation sequencing
-
Paruzynski A, Arens A, Gabriel R, Bartholomae CC, Scholz S, et al. (2010) Genome-wide high-throughput integrome analyses by nrLAM-PCR and next-generation sequencing. Nat Protoc 5: 1379-1395.
-
(2010)
Nat Protoc
, vol.5
, pp. 1379-1395
-
-
Paruzynski, A.1
Arens, A.2
Gabriel, R.3
Bartholomae, C.C.4
Scholz, S.5
-
21
-
-
36749052217
-
High-resolution insertion-site analysis by linear amplification-mediated PCR (LAM-PCR)
-
Schmidt M, Schwarzwaelder K, Bartholomae C, Zaoui K, Ball C, et al. (2007) High-resolution insertion-site analysis by linear amplification-mediated PCR (LAM-PCR). Nat Methods 4: 1051-1057.
-
(2007)
Nat Methods
, vol.4
, pp. 1051-1057
-
-
Schmidt, M.1
Schwarzwaelder, K.2
Bartholomae, C.3
Zaoui, K.4
Ball, C.5
-
22
-
-
0036729878
-
High-throughput retroviral tagging to identify components of specific signaling pathways in cancer
-
Mikkers H, Allen J, Knipscheer P, Romeijn L, Hart A, et al. (2002) High-throughput retroviral tagging to identify components of specific signaling pathways in cancer. Nat Genet 32: 153-159.
-
(2002)
Nat Genet
, vol.32
, pp. 153-159
-
-
Mikkers, H.1
Allen, J.2
Knipscheer, P.3
Romeijn, L.4
Hart, A.5
-
23
-
-
0036725322
-
Genome-wide retroviral insertional tagging of genes involved in cancer in Cdkn2a-deficient mice
-
Lund AH, Turner G, Trubetskoy A, Verhoeven E, Wientjens E, et al. (2002) Genome-wide retroviral insertional tagging of genes involved in cancer in Cdkn2a-deficient mice. Nat Genet 32: 160-165.
-
(2002)
Nat Genet
, vol.32
, pp. 160-165
-
-
Lund, A.H.1
Turner, G.2
Trubetskoy, A.3
Verhoeven, E.4
Wientjens, E.5
-
24
-
-
0036725051
-
New genes involved in cancer identified by retroviral tagging
-
Suzuki T, Shen H, Akagi K, Morse HC, Malley JD, et al. (2002) New genes involved in cancer identified by retroviral tagging. Nat Genet 32: 166-174.
-
(2002)
Nat Genet
, vol.32
, pp. 166-174
-
-
Suzuki, T.1
Shen, H.2
Akagi, K.3
Morse, H.C.4
Malley, J.D.5
-
25
-
-
0037162715
-
HIV-1 integration in the human genome favors active genes and local hotspots
-
Schroder AR, Shinn P, Chen H, Berry C, Ecker JR, et al. (2002) HIV-1 integration in the human genome favors active genes and local hotspots. Cell 110: 521-529.
-
(2002)
Cell
, vol.110
, pp. 521-529
-
-
Schroder, A.R.1
Shinn, P.2
Chen, H.3
Berry, C.4
Ecker, J.R.5
-
26
-
-
19344375031
-
Retroviral DNA integration: ASLV, HIV, and MLV show distinct target site preferences
-
Mitchell RS, Beitzel BF, Schroder AR, Shinn P, Chen H, et al. (2004) Retroviral DNA integration: ASLV, HIV, and MLV show distinct target site preferences. PLoS Biol 2: E234.
-
(2004)
PLoS Biol
, vol.2
-
-
Mitchell, R.S.1
Beitzel, B.F.2
Schroder, A.R.3
Shinn, P.4
Chen, H.5
-
27
-
-
33846025793
-
Detecting statistically significant common insertion sites in retroviral insertional mutagenesis screens
-
de Ridder J, Uren A, Kool J, Reinders M, Wessels L, (2006) Detecting statistically significant common insertion sites in retroviral insertional mutagenesis screens. PLoS Comput Biol 2: e166.
-
(2006)
PLoS Comput Biol
, vol.2
-
-
de Ridder, J.1
Uren, A.2
Kool, J.3
Reinders, M.4
Wessels, L.5
-
28
-
-
63049105067
-
A Transposon-Based Genetic Screen in Mice Identifies Genes Altered in Colorectal Cancer
-
Starr TK, Allaei R, Silverstein KAT, Staggs RA, Sarver AL, et al. (2009) A Transposon-Based Genetic Screen in Mice Identifies Genes Altered in Colorectal Cancer. Science 323: 1747-1750.
-
(2009)
Science
, vol.323
, pp. 1747-1750
-
-
Starr, T.K.1
Allaei, R.2
Silverstein, K.A.T.3
Staggs, R.A.4
Sarver, A.L.5
-
29
-
-
70449365607
-
Confidence interval for the bootstrap P-value and sample size calculation of the bootstrap test
-
Li J, Tai BC, Nott DJ, (2009) Confidence interval for the bootstrap P-value and sample size calculation of the bootstrap test. Journal of Nonparametric Statistics 21: 649-661.
-
(2009)
Journal of Nonparametric Statistics
, vol.21
, pp. 649-661
-
-
Li, J.1
Tai, B.C.2
Nott, D.J.3
-
30
-
-
0003488728
-
Multiple Comparisons
-
2 Ed, London, Chapman & Hall
-
Hsu JC, (1996) Multiple comparisons. Theory and methods. 2 ed London Chapman & Hall.
-
(1996)
Theory and Methods
-
-
Hsu, J.C.1
-
31
-
-
37749008710
-
Real-time definition of non-randomness in the distribution of genomic events
-
Abel U, Deichmann A, Bartholomae C, Schwarzwaelder K, Glimm H, et al. (2007) Real-time definition of non-randomness in the distribution of genomic events. PLoS ONE 2: e570.
-
(2007)
PLoS ONE
, vol.2
-
-
Abel, U.1
Deichmann, A.2
Bartholomae, C.3
Schwarzwaelder, K.4
Glimm, H.5
-
33
-
-
30044442930
-
Redefining the common insertion site
-
Wu X, Luke BT, Burgess SM, (2006) Redefining the common insertion site. Virology 344: 292-295.
-
(2006)
Virology
, vol.344
, pp. 292-295
-
-
Wu, X.1
Luke, B.T.2
Burgess, S.M.3
-
34
-
-
79953296630
-
Lentiviral Vector Integration Profiles Differ in Rodent Postmitotic Tissues
-
Bartholomae CC, Arens A, Balaggan KS, Yanez-Munoz RJ, Montini E, et al. (2011) Lentiviral Vector Integration Profiles Differ in Rodent Postmitotic Tissues. Mol Ther 19: 703-710.
-
(2011)
Mol Ther
, vol.19
, pp. 703-710
-
-
Bartholomae, C.C.1
Arens, A.2
Balaggan, K.S.3
Yanez-Munoz, R.J.4
Montini, E.5
-
35
-
-
84872639295
-
Significance tests for 2×2 tables
-
Barnard GA, (1947) Significance tests for 2×2 tables. Biometrika 34: 123-138.
-
(1947)
Biometrika
, vol.34
, pp. 123-138
-
-
Barnard, G.A.1
-
36
-
-
70449427834
-
Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy
-
Cartier N, Hacein-Bey-Abina S, Bartholomae CC, Veres G, Schmidt M, et al. (2009) Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy. Science 326: 818-823.
-
(2009)
Science
, vol.326
, pp. 818-823
-
-
Cartier, N.1
Hacein-Bey-Abina, S.2
Bartholomae, C.C.3
Veres, G.4
Schmidt, M.5
|