-
1
-
-
0002900077
-
Gene therapy for hematopoietic diseases
-
BP Sorrentino AW Nienhuis Gene therapy for hematopoietic diseases G Stamatoyannopoulous PW Majerus RM Perlmutter H Varmus The Molecular Basis of Blood Diseases 3rd edn 2001 Saunders Philadelphia, PA 969 1003
-
(2001)
, pp. 969-1003
-
-
Sorrentino, BP1
Nienhuis, AW2
-
3
-
-
19944421384
-
Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector
-
HB Gaspar KL Parsley S Howe D King KC Gilmour J Sinclair Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector Lancet 364 2004 2181 2187
-
(2004)
Lancet
, vol.364
, pp. 2181-2187
-
-
Gaspar, HB1
Parsley, KL2
Howe, S3
King, D4
Gilmour, KC5
Sinclair, J6
-
5
-
-
34547118806
-
What are the consequences of the fourth case?
-
C Baum What are the consequences of the fourth case? Mol Ther 15 2007 1401 1402
-
(2007)
Mol Ther
, vol.15
, pp. 1401-1402
-
-
Baum, C1
-
6
-
-
0042738935
-
Occurrence of leukaemia following gene therapy of X-linked SCID
-
DB Kohn M Sadelain JC Glorioso Occurrence of leukaemia following gene therapy of X-linked SCID Nat Rev Cancer 3 2003 477 488
-
(2003)
Nat Rev Cancer
, vol.3
, pp. 477-488
-
-
Kohn, DB1
Sadelain, M2
Glorioso, JC3
-
7
-
-
34249906359
-
Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy
-
A Deichmann S Hacein-Bey-Abina M Schmidt A Garrigue MH Brugman J Hu Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy J Clin Invest 117 2007 2225 2232
-
(2007)
J Clin Invest
, vol.117
, pp. 2225-2232
-
-
Deichmann, A1
Hacein-Bey-Abina, S2
Schmidt, M3
Garrigue, A4
Brugman, MH5
Hu, J6
-
8
-
-
34547651095
-
Gammaretrovirus-mediated correction of SCID-X1 is associated with skewed vector integration site distribution in vivo
-
K Schwarzwaelder SJ Howe M Schmidt MH Brugman A Deichmann H Glimm Gammaretrovirus-mediated correction of SCID-X1 is associated with skewed vector integration site distribution in vivo J Clin Invest 117 2007 2241 2249
-
(2007)
J Clin Invest
, vol.117
, pp. 2241-2249
-
-
Schwarzwaelder, K1
Howe, SJ2
Schmidt, M3
Brugman, MH4
Deichmann, A5
Glimm, H6
-
9
-
-
0023678897
-
Retroviral activation of a novel gene encoding a zinc finger protein in IL-3-dependent myeloid leukemia cell lines
-
K Morishita DS Parker ML Mucenski NA Jenkins NG Copeland JN Ihle Retroviral activation of a novel gene encoding a zinc finger protein in IL-3-dependent myeloid leukemia cell lines Cell 54 1988 831 840
-
(1988)
Cell
, vol.54
, pp. 831-840
-
-
Morishita, K1
Parker, DS2
Mucenski, ML3
Jenkins, NA4
Copeland, NG5
Ihle, JN6
-
11
-
-
27944449787
-
Insertional mutagenesis identifies genes that promote the immortalization of primary bone marrow progenitor cells
-
Y Du NA Jenkins NG Copeland Insertional mutagenesis identifies genes that promote the immortalization of primary bone marrow progenitor cells Blood 106 2005 3932 3939
-
(2005)
Blood
, vol.106
, pp. 3932-3939
-
-
Du, Y1
Jenkins, NA2
Copeland, NG3
-
12
-
-
33750624015
-
Cell-culture assays reveal the importance of retroviral vector design for insertional genotoxicity
-
U Modlich J Bohne M Schmidt C von Kalle S Knöss A Schambach Cell-culture assays reveal the importance of retroviral vector design for insertional genotoxicity Blood 108 2006 2545 2553
-
(2006)
Blood
, vol.108
, pp. 2545-2553
-
-
Modlich, U1
Bohne, J2
Schmidt, M3
von Kalle, C4
Knöss, S5
Schambach, A6
-
13
-
-
21044448037
-
Clonal dominance of hematopoietic stem cells triggered by retroviral gene marking
-
O Kustikova B Fehse U Modlich M Yang J Düllmann K Kamino Clonal dominance of hematopoietic stem cells triggered by retroviral gene marking Science 308 2005 1171 1174
-
(2005)
Science
, vol.308
, pp. 1171-1174
-
-
Kustikova, O1
Fehse, B2
Modlich, U3
Yang, M4
Düllmann, J5
Kamino, K6
-
15
-
-
25144490586
-
Recurrent retroviral vector integration at the Mds1/Evi1 locus in nonhuman primate hematopoietic cells
-
B Calmels C Ferguson MO Laukkanen R Adler M Faulhaber HJ Kim Recurrent retroviral vector integration at the Mds1/Evi1 locus in nonhuman primate hematopoietic cells Blood 106 2005 2530 2533
-
(2005)
Blood
, vol.106
, pp. 2530-2533
-
-
Calmels, B1
Ferguson, C2
Laukkanen, MO3
Adler, R4
Faulhaber, M5
Kim, HJ6
-
16
-
-
33646594123
-
Acute myeloid leukemia associated with retroviral gene transfer to hematopoietic progenitor cells in a rhesus macaque
-
R Seggewiss S Pittaluga RL Adler FJ Guenaga C Ferguson IH Pilz Acute myeloid leukemia associated with retroviral gene transfer to hematopoietic progenitor cells in a rhesus macaque Blood 107 2006 3865 3867
-
(2006)
Blood
, vol.107
, pp. 3865-3867
-
-
Seggewiss, R1
Pittaluga, S2
Adler, RL3
Guenaga, FJ4
Ferguson, C5
Pilz, IH6
-
17
-
-
33645734405
-
Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1
-
MG Ott M Schmidt K Schwarzwaelder S Stein U Siler U Koehl Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1 Nat Med 12 2006 401 409
-
(2006)
Nat Med
, vol.12
, pp. 401-409
-
-
Ott, MG1
Schmidt, M2
Schwarzwaelder, K3
Stein, S4
Siler, U5
Koehl, U6
-
18
-
-
0343628721
-
Therapeutic haemoglobin synthesis in β-thalassaemic mice expressing lentivirus-encoded human β-globin
-
C May S Rivella J Callegari G Heller KM Gaensler L Luzzatto Therapeutic haemoglobin synthesis in β -thalassaemic mice expressing lentivirus-encoded human β -globin Nature 406 2000 82 86
-
(2000)
Nature
, vol.406
, pp. 82-86
-
-
May, C1
Rivella, S2
Callegari, J3
Heller, G4
Gaensler, KM5
Luzzatto, L6
-
20
-
-
0037443467
-
The degree of phenotypic correction of murine β-thalassemia intermedia following lentiviral-mediated transfer of a human γ-globin gene is influenced by chromosomal position effects and vector copy number
-
DA Persons PW Hargrove ER Allay H Hanawa AW Nienhuis The degree of phenotypic correction of murine β -thalassemia intermedia following lentiviral-mediated transfer of a human γ -globin gene is influenced by chromosomal position effects and vector copy number Blood 101 2003 2175 2183
-
(2003)
Blood
, vol.101
, pp. 2175-2183
-
-
Persons, DA1
Hargrove, PW2
Allay, ER3
Hanawa, H4
Nienhuis, AW5
-
21
-
-
0037195091
-
Permanent and panerythroid correction of murine β-thalassemia by multiple lentiviral integration in hematopoietic stem cells
-
S Imren E Payen KA Westerman R Pawliuk ME Fabry CJ Eaves Permanent and panerythroid correction of murine β -thalassemia by multiple lentiviral integration in hematopoietic stem cells Proc Natl Acad Sci USA 99 2002 14380 14385
-
(2002)
Proc Natl Acad Sci USA
, vol.99
, pp. 14380-14385
-
-
Imren, S1
Payen, E2
Westerman, KA3
Pawliuk, R4
Fabry, ME5
Eaves, CJ6
-
22
-
-
0344305491
-
Correction of a mouse model of sickle cell disease: lentiviral/antisickling β-globin gene transduction of unmobilized, purified hematopoietic stem cells
-
DN Levasseur TM Ryan KM Pawlik TM Townes Correction of a mouse model of sickle cell disease: lentiviral/antisickling β -globin gene transduction of unmobilized, purified hematopoietic stem cells Blood 102 2003 4312 4319
-
(2003)
Blood
, vol.102
, pp. 4312-4319
-
-
Levasseur, DN1
Ryan, TM2
Pawlik, KM3
Townes, TM4
-
23
-
-
33646887376
-
Genotoxicity of retroviral integration in hematopoietic cells
-
AW Nienhuis CE Dunbar BP Sorrentino Genotoxicity of retroviral integration in hematopoietic cells Mol Ther 13 2006 1031 1049
-
(2006)
Mol Ther
, vol.13
, pp. 1031-1049
-
-
Nienhuis, AW1
Dunbar, CE2
Sorrentino, BP3
-
24
-
-
33847205752
-
The genetic engineering of hematopoietic stem cells: the rise of lentiviral vectors, the conundrum of the LTR, and the promise of lineage-restricted vectors
-
AH Chang M Sadelain The genetic engineering of hematopoietic stem cells: the rise of lentiviral vectors, the conundrum of the LTR, and the promise of lineage-restricted vectors Mol Ther 15 2007 445 456
-
(2007)
Mol Ther
, vol.15
, pp. 445-456
-
-
Chang, AH1
Sadelain, M2
-
25
-
-
0037162715
-
HIV-1 integration in the human genome favors active genes and local hotspots
-
AR Schröder P Shinn H Chen C Berry Jr Ecker F Bushman 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
-
-
Schröder, AR1
Shinn, P2
Chen, H3
Berry, C4
Ecker, Jr5
Bushman, F6
-
26
-
-
0037841763
-
Transcription start regions in the human genome are favored targets for MLV integration
-
X Wu Y Li B Crise SM 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, X1
Li, Y2
Crise, B3
Burgess, SM4
-
27
-
-
20044396722
-
Distinct genomic integration of MLV and SIV vectors in primate hematopoietic stem and progenitor cells
-
P Hematti BK Hong C Ferguson R Adler H Hanawa S Sellers Distinct genomic integration of MLV and SIV vectors in primate hematopoietic stem and progenitor cells PLoS Biol 2 2003 e423
-
(2003)
PLoS Biol
, vol.2
, pp. e423
-
-
Hematti, P1
Hong, BK2
Ferguson, C3
Adler, R4
Hanawa, H5
Sellers, S6
-
28
-
-
33745108790
-
Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration
-
E Montini D Cesana M Schmidt F Sanvito M Ponzoni C Bartholomae Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration Nat Biotechnol 24 2006 687 696
-
(2006)
Nat Biotechnol
, vol.24
, pp. 687-696
-
-
Montini, E1
Cesana, D2
Schmidt, M3
Sanvito, F4
Ponzoni, M5
Bartholomae, C6
-
29
-
-
9644291600
-
High-level β-globin expression and preferred intragenic integration after lentiviral transduction of human cord blood stem cells
-
S Imren ME Fabry KA Westerman R Pawliuk P Tang PM Rosten High-level β -globin expression and preferred intragenic integration after lentiviral transduction of human cord blood stem cells J Clin Invest 114 2004 953 962
-
(2004)
J Clin Invest
, vol.114
, pp. 953-962
-
-
Imren, S1
Fabry, ME2
Westerman, KA3
Pawliuk, R4
Tang, P5
Rosten, PM6
-
30
-
-
0037441587
-
Lentiviral vector transduction of NOD/SCID repopulating cells results in multiple vector integrations per transduced cell: risk of insertional mutagenesis
-
NB Woods A Muessig M Schmidt J Flygare K Olsson P Salmon Lentiviral vector transduction of NOD/SCID repopulating cells results in multiple vector integrations per transduced cell: risk of insertional mutagenesis Blood 101 2003 1284 1289
-
(2003)
Blood
, vol.101
, pp. 1284-1289
-
-
Woods, NB1
Muessig, A2
Schmidt, M3
Flygare, J4
Olsson, K5
Salmon, P6
-
31
-
-
0020052387
-
Transient nature of early haematopoietic spleen colonies
-
MC Magli NN Iscove N Odartchenko Transient nature of early haematopoietic spleen colonies Nature 295 1982 527 529
-
(1982)
Nature
, vol.295
, pp. 527-529
-
-
Magli, MC1
Iscove, NN2
Odartchenko, N3
-
32
-
-
4944234406
-
Extended β-globin locus control region elements promote consistent therapeutic expression of a γ-globin lentiviral vector in murine β-thalassemia
-
H Hanawa PW Hargrove S Kepes DK Srivastava AW Nienhuis DA Persons Extended β -globin locus control region elements promote consistent therapeutic expression of a γ -globin lentiviral vector in murine β -thalassemia Blood 104 2004 2281 2290
-
(2004)
Blood
, vol.104
, pp. 2281-2290
-
-
Hanawa, H1
Hargrove, PW2
Kepes, S3
Srivastava, DK4
Nienhuis, AW5
Persons, DA6
-
33
-
-
8644228649
-
Global regulation of erythroid gene expression by transcription factor GATA-1
-
JJ Welch JA Watts CR Vakoc Y Yao H Wang RC Hardison Global regulation of erythroid gene expression by transcription factor GATA-1 Blood 104 2004 3136 3147
-
(2004)
Blood
, vol.104
, pp. 3136-3147
-
-
Welch, JJ1
Watts, JA2
Vakoc, CR3
Yao, Y4
Wang, H5
Hardison, RC6
-
34
-
-
19344371219
-
The zebrafish moonshine gene encodes transcriptional intermediary factor 1γ, an essential regulator of hematopoiesis
-
DG Ransom N Bahary K Niss D Traver C Burns NS Trede The zebrafish moonshine gene encodes transcriptional intermediary factor 1 γ , an essential regulator of hematopoiesis PLoS Biol 2 2004 E237
-
(2004)
PLoS Biol
, vol.2
, pp. E237
-
-
Ransom, DG1
Bahary, N2
Niss, K3
Traver, D4
Burns, C5
Trede, NS6
-
35
-
-
34249804819
-
Unique integration profiles in a canine model of long-term repopulating cells transduced with gammaretrovirus, lentivirus, or foamy virus
-
KA Keyser BC Beard G Trobridge LJ Peterson D Miller M Jacobs Unique integration profiles in a canine model of long-term repopulating cells transduced with gammaretrovirus, lentivirus, or foamy virus Hum Gene Ther 8 2007 423 434
-
(2007)
Hum Gene Ther
, vol.8
, pp. 423-434
-
-
Keyser, KA1
Beard, BC2
Trobridge, G3
Peterson, LJ4
Miller, D5
Jacobs, M6
-
36
-
-
24644482422
-
Reduced PU.1 expression causes myeloid progenitor expansion and increased leukemia penetrance in mice expressing PML-RARα
-
MJ Walter JS Park RE Ries SK Lau M McLellan S Jaeger Reduced PU.1 expression causes myeloid progenitor expansion and increased leukemia penetrance in mice expressing PML-RAR α Proc Natl Acad Sci USA 102 2005 12513 12518
-
(2005)
Proc Natl Acad Sci USA
, vol.102
, pp. 12513-12518
-
-
Walter, MJ1
Park, JS2
Ries, RE3
Lau, SK4
McLellan, M5
Jaeger, S6
-
37
-
-
0029091845
-
Overexpression of HOXB4 in hematopoietic cells causes the selective expansion of more primitive populations in vitro and in vivo
-
G Sauvageau U Thorsteinsdottir CJ Eaves HJ Lawrence C Largman PM Lansdorp Overexpression of HOXB4 in hematopoietic cells causes the selective expansion of more primitive populations in vitro and in vivo Genes Dev 9 1995 1753 1765
-
(1995)
Genes Dev
, vol.9
, pp. 1753-1765
-
-
Sauvageau, G1
Thorsteinsdottir, U2
Eaves, CJ3
Lawrence, HJ4
Largman, C5
Lansdorp, PM6
-
38
-
-
31944440000
-
Retroviral vector integration deregulates gene expression but has no consequence on the biology and function of transplanted T cells
-
A Recchia C Bonini Z Magnani F Urbinati D Sartori S Muraro Retroviral vector integration deregulates gene expression but has no consequence on the biology and function of transplanted T cells Proc Natl Acad Sci USA 103 2006 1457 1462
-
(2006)
Proc Natl Acad Sci USA
, vol.103
, pp. 1457-1462
-
-
Recchia, A1
Bonini, C2
Magnani, Z3
Urbinati, F4
Sartori, D5
Muraro, S6
-
39
-
-
29444448047
-
On a chromosome far, far away: LCRs and gene expression
-
A Dean On a chromosome far, far away: LCRs and gene expression Trends Genet 22 2006 38 45
-
(2006)
Trends Genet
, vol.22
, pp. 38-45
-
-
Dean, A1
-
41
-
-
0023839919
-
Spi-1 is a putative oncogene in virally induced murine erythroleukaemias
-
F Moreau-Gachelin A Tavitian P Tambourin Spi-1 is a putative oncogene in virally induced murine erythroleukaemias Nature 331 1988 277 280
-
(1988)
Nature
, vol.331
, pp. 277-280
-
-
Moreau-Gachelin, F1
Tavitian, A2
Tambourin, P3
-
43
-
-
0026326555
-
An activating mutation in the murine erythropoietin receptor induces erythroleukemia in mice: a cytokine receptor superfamily oncogene
-
GD Longmore HF Lodish An activating mutation in the murine erythropoietin receptor induces erythroleukemia in mice: a cytokine receptor superfamily oncogene Cell 67 1991 1089 1102
-
(1991)
Cell
, vol.67
, pp. 1089-1102
-
-
Longmore, GD1
Lodish, HF2
-
44
-
-
0034554856
-
Friend erythroleukemia revisited
-
PA Ney AD D'Andrea Friend erythroleukemia revisited Blood 96 2000 3675 3680
-
(2000)
Blood
, vol.96
, pp. 3675-3680
-
-
Ney, PA1
D'Andrea, AD2
-
45
-
-
28844501416
-
Kit-activating mutations cooperate with Spi-1/PU.1 overexpression to promote tumorigenic progression during erythroleukemia in mice
-
O Kosmider N Denis C Lacout W Vainchenker P Dubreuil F Moreau-Gachelin Kit-activating mutations cooperate with Spi-1/PU.1 overexpression to promote tumorigenic progression during erythroleukemia in mice Cancer Cell 8 2005 467 478
-
(2005)
Cancer Cell
, vol.8
, pp. 467-478
-
-
Kosmider, O1
Denis, N2
Lacout, C3
Vainchenker, W4
Dubreuil, P5
Moreau-Gachelin, F6
-
46
-
-
0034651726
-
Hemopoietic lineage commitment decisions: in vivo evidence from a transgenic mouse model harboring micro LCR-β pro-LacZ as a transgene
-
T Papayannopoulou GV Priestley A Rohde KR Peterson B Nakamotos Hemopoietic lineage commitment decisions: in vivo evidence from a transgenic mouse model harboring micro LCR- β pro-LacZ as a transgene Blood 95 2000 1274 1282
-
(2000)
Blood
, vol.95
, pp. 1274-1282
-
-
Papayannopoulou, T1
Priestley, GV2
Rohde, A3
Peterson, KR4
Nakamotos, B5
-
47
-
-
0036446597
-
High-level erythroid lineage-directed gene expression using globin gene regulatory elements after lentiviral vector-mediated gene transfer into primitive human and murine hematopoietic cells
-
H Hanawa DA Persons AW Nienhuis High-level erythroid lineage-directed gene expression using globin gene regulatory elements after lentiviral vector-mediated gene transfer into primitive human and murine hematopoietic cells Hum Gene Ther 13 2002 2007 2016
-
(2002)
Hum Gene Ther
, vol.13
, pp. 2007-2016
-
-
Hanawa, H1
Persons, DA2
Nienhuis, AW3
-
49
-
-
34748902850
-
A chromatin insulator blocks interactions between globin regulatory elements and cellular promoters in erythroid cells
-
BY Ryu DA Persons MV Evans-Galea JT Gray AW Nienhuis A chromatin insulator blocks interactions between globin regulatory elements and cellular promoters in erythroid cells Blood Cells Mol Dis 39 2007 221 228
-
(2007)
Blood Cells Mol Dis
, vol.39
, pp. 221-228
-
-
Ryu, BY1
Persons, DA2
Evans-Galea, MV3
Gray, JT4
Nienhuis, AW5
-
50
-
-
33847408501
-
Retroviral vector insertion sites associated with dominant hematopoietic clones mark “stemness” pathways
-
OS Kustikova H Geiger Z Li MH Brugman SM Chambers CA Shaw Retroviral vector insertion sites associated with dominant hematopoietic clones mark “stemness” pathways Blood 109 2007 1897 1907
-
(2007)
Blood
, vol.109
, pp. 1897-1907
-
-
Kustikova, OS1
Geiger, H2
Li, Z3
Brugman, MH4
Chambers, SM5
Shaw, CA6
|