-
3
-
-
0035320886
-
Phenotype-genotype relationships in monogenic disease: Lessons from the thalassaemias
-
Weatherall DJ. Phenotype-genotype relationships in monogenic disease: lessons from the thalassaemias. Nat Rev Genet 2001; 2:245-255.
-
(2001)
Nat Rev Genet
, vol.2
, pp. 245-255
-
-
Weatherall, D.J.1
-
5
-
-
0002341637
-
A series of cases of splenomegaly in children with anemia and peculiar bone changes
-
Cooley TB, Lee P. A series of cases of splenomegaly in children with anemia and peculiar bone changes. Trans Am Pediatr Soc 1925; 37:29.
-
(1925)
Trans Am Pediatr Soc
, vol.37
, pp. 29
-
-
Cooley, T.B.1
Lee, P.2
-
6
-
-
0034783438
-
Chelation therapy in beta-thalassemia: An optimistic update
-
Giardina PJ, Grady RW. Chelation therapy in beta-thalassemia: an optimistic update. Semin Hematol 2001; 38:360-366.
-
(2001)
Semin Hematol
, vol.38
, pp. 360-366
-
-
Giardina, P.J.1
Grady, R.W.2
-
7
-
-
0028401662
-
Bone marrow transplantation in the treatment of thalassemia
-
Giardini C, Lucarelli G. Bone marrow transplantation in the treatment of thalassemia. Curr Opin Hematol 1994; 1:170-176.
-
(1994)
Curr Opin Hematol
, vol.1
, pp. 170-176
-
-
Giardini, C.1
Lucarelli, G.2
-
8
-
-
0031824758
-
Bone marrow transplantation for homozygous beta-thalassemia. The Memorial Sloan-Kettering Cancer Center experience
-
Boulad F, Giardina P, Gillio A, et al. Bone marrow transplantation for homozygous beta-thalassemia. The Memorial Sloan-Kettering Cancer Center experience. Ann N Y Acad Sci 1998; 850:498-502.
-
(1998)
Ann N Y Acad Sci
, vol.850
, pp. 498-502
-
-
Boulad, F.1
Giardina, P.2
Gillio, A.3
-
9
-
-
0033558264
-
Bone marrow transplantation in adult thalassemic patients
-
Lucarelli G, Clift RA, Galimberti M, et al. Bone marrow transplantation in adult thalassemic patients. Blood 1999; 93:1164-1167.
-
(1999)
Blood
, vol.93
, pp. 1164-1167
-
-
Lucarelli, G.1
Clift, R.A.2
Galimberti, M.3
-
10
-
-
0034783710
-
Toward gene therapy for disorders of globin synthesis
-
Tisdale J, Sadelain M. Toward gene therapy for disorders of globin synthesis. Semin Hematol 2001; 38:382-392.
-
(2001)
Semin Hematol
, vol.38
, pp. 382-392
-
-
Tisdale, J.1
Sadelain, M.2
-
11
-
-
0000844285
-
Molecular mechanism of β thalassemia
-
Steinberg MH, Forget BG, Higgs DR, Nagel RL, editors. Cambridge, UK: Cambridge University Press
-
Forget BG. Molecular mechanism of β thalassemia. In: Steinberg MH, Forget BG, Higgs DR, Nagel RL, editors. Disorders of haemoglobin: genetics, pathophysiology, and clinical management. Cambridge, UK: Cambridge University Press; 2001. pp. 252-276.
-
(2001)
Disorders of Haemoglobin: Genetics, Pathophysiology, and Clinical Management
, pp. 252-276
-
-
Forget, B.G.1
-
12
-
-
0000192486
-
Sickle cell anemia, a molecular disease
-
Pauling L, Itano HA, Singer SJ, Wells IC. Sickle cell anemia, a molecular disease. Science 1949; 110:543-546.
-
(1949)
Science
, vol.110
, pp. 543-546
-
-
Pauling, L.1
Itano, H.A.2
Singer, S.J.3
Wells, I.C.4
-
14
-
-
0021286875
-
Hydroxyurea enhances fetal hemoglobin production in sickle cell anemia
-
Platt OS, Orkin SH, Dover G, et al. Hydroxyurea enhances fetal hemoglobin production in sickle cell anemia. J Clin Invest 1984; 74:652-656.
-
(1984)
J Clin Invest
, vol.74
, pp. 652-656
-
-
Platt, O.S.1
Orkin, S.H.2
Dover, G.3
-
15
-
-
0026631318
-
Hydroxyurea: Effects on hemoglobin F production in patients with sickle cell anemia
-
Charache S, Dover GJ, Moore RD, et al. Hydroxyurea: effects on hemoglobin F production in patients with sickle cell anemia. Blood 1992; 79:2555-2565.
-
(1992)
Blood
, vol.79
, pp. 2555-2565
-
-
Charache, S.1
Dover, G.J.2
Moore, R.D.3
-
16
-
-
0034772589
-
Pharmacological induction of fetal hemoglobin in sickle cell disease and beta-thalassemia
-
Atweh GF, Loukopoulos D. Pharmacological induction of fetal hemoglobin in sickle cell disease and beta-thalassemia. Semin Hematol 2001; 38:367-373.
-
(2001)
Semin Hematol
, vol.38
, pp. 367-373
-
-
Atweh, G.F.1
Loukopoulos, D.2
-
17
-
-
29744433240
-
Induction of fetal globin in β-thalassemia: Cellular obstacles and molecular progress
-
Perrine SP, Castaneda SA, Boosalis MS, et al. Induction of fetal globin in β-thalassemia: cellular obstacles and molecular progress. Ann N Y Acad Sci 2005; 1054:257-265.
-
(2005)
Ann N Y Acad Sci
, vol.1054
, pp. 257-265
-
-
Perrine, S.P.1
Castaneda, S.A.2
Boosalis, M.S.3
-
18
-
-
29244432479
-
Prospects for developing a molecular cure for thalassemia
-
Stamatoyannopoulos G. Prospects for developing a molecular cure for thalassemia. Hematology 2005; 10 (Suppl 1):255-257.
-
(2005)
Hematology
, vol.10
, Issue.1 SUPPL.
, pp. 255-257
-
-
Stamatoyannopoulos, G.1
-
19
-
-
0031870487
-
Haematopoietic stem cell transplantation for sickle cell anaemia: The first 50 patients transplanted in Belgium
-
Vermylen C, Cornu G, Ferster A, et al. Haematopoietic stem cell transplantation for sickle cell anaemia: the first 50 patients transplanted in Belgium. Bone Marrow Transplant 1998; 22:1-6.
-
(1998)
Bone Marrow Transplant
, vol.22
, pp. 1-6
-
-
Vermylen, C.1
Cornu, G.2
Ferster, A.3
-
20
-
-
0024961622
-
Sickle cell anaemia: A simple disease with no cure
-
Luzzatto L, Goodfellow P. Sickle cell anaemia: a simple disease with no cure. Nature 1989; 337:17-18.
-
(1989)
Nature
, vol.337
, pp. 17-18
-
-
Luzzatto, L.1
Goodfellow, P.2
-
21
-
-
0030810446
-
Genetic treatment of the haemoglobinopathies: Recombinations and new combinations
-
Sadelain M. Genetic treatment of the haemoglobinopathies: recombinations and new combinations. Br J Haematol 1997; 98:247-253.
-
(1997)
Br J Haematol
, vol.98
, pp. 247-253
-
-
Sadelain, M.1
-
22
-
-
0028303758
-
Mutagenesis of retroviral vectors transducing human beta-globin gene and beta-globin locus control region derivatives results in stable transmission of an active transcriptional structure
-
Leboulch P, Huang GM, Humphries RK, et al. Mutagenesis of retroviral vectors transducing human beta-globin gene and beta-globin locus control region derivatives results in stable transmission of an active transcriptional structure. EMBO J 1994; 13:3065-3076.
-
(1994)
EMBO J
, vol.13
, pp. 3065-3076
-
-
Leboulch, P.1
Huang, G.M.2
Humphries, R.K.3
-
23
-
-
0029084785
-
Generation of a high-titer retroviral vector capable of expressing high levels of the human beta-globin gene
-
U S A
-
Sadelain M, Wang CH, Antoniou M, et al. Generation of a high-titer retroviral vector capable of expressing high levels of the human beta-globin gene. Proc Natl Acad Sci U S A 1995; 92:6728-6732.
-
(1995)
Proc Natl Acad Sci
, vol.92
, pp. 6728-6732
-
-
Sadelain, M.1
Wang, C.H.2
Antoniou, M.3
-
24
-
-
0023113052
-
Regulated expression of a complete human beta-globin gene encoded by a transmissible retrovirus vector
-
Cone RD, Weber-Benarous A, Baorto D, Mulligan RC. Regulated expression of a complete human beta-globin gene encoded by a transmissible retrovirus vector. Mol Cell Biol 1987; 7:887-897.
-
(1987)
Mol Cell Biol
, vol.7
, pp. 887-897
-
-
Cone, R.D.1
Weber-Benarous, A.2
Baorto, D.3
Mulligan, R.C.4
-
25
-
-
0023191239
-
Retroviral-mediated transfer of genomic globin genes leads to regulated production of RNA and protein
-
U S A
-
Karlsson S, Papayannopoulou T, Schweiger SG, et al. Retroviral-mediated transfer of genomic globin genes leads to regulated production of RNA and protein. Proc Natl Acad Sci U S A 1987; 84:2411-2415.
-
(1987)
Proc Natl Acad Sci
, vol.84
, pp. 2411-2415
-
-
Karlsson, S.1
Papayannopoulou, T.2
Schweiger, S.G.3
-
26
-
-
0023864651
-
Lineage-specific expression of a human beta-globin gene in murine bone marrow transplant recipients reconstituted with retrovirus-transduced stem cells
-
Dzierzak EA, Papayannopoulou T, Mulligan RC. Lineage-specific expression of a human beta-globin gene in murine bone marrow transplant recipients reconstituted with retrovirus-transduced stem cells. Nature 1988; 331:35-41.
-
(1988)
Nature
, vol.331
, pp. 35-41
-
-
Dzierzak, E.A.1
Papayannopoulou, T.2
Mulligan, R.C.3
-
27
-
-
0024505741
-
A majority of mice show long-term expression of a human beta-globin gene after retrovirus transfer into hematopoietic stem cells
-
Bender MA, Gelinas RE, Miller AD. A majority of mice show long-term expression of a human beta-globin gene after retrovirus transfer into hematopoietic stem cells. Mol Cell Biol 1989; 9:1426-1434.
-
(1989)
Mol Cell Biol
, vol.9
, pp. 1426-1434
-
-
Bender, M.A.1
Gelinas, R.E.2
Miller, A.D.3
-
28
-
-
0024380889
-
Combination of interleukins 3 and 6 preserves stem cell function in culture and enhances retrovirus-mediated gene transfer into hematopoietic stem cells
-
USA
-
Bodine DM, Karlsson S, Nienhuis AW. Combination of interleukins 3 and 6 preserves stem cell function in culture and enhances retrovirus-mediated gene transfer into hematopoietic stem cells. Proc Natl Acad Sci USA 1989; 86:8897-8901.
-
(1989)
Proc Natl Acad Sci
, vol.86
, pp. 8897-8901
-
-
Bodine, D.M.1
Karlsson, S.2
Nienhuis, A.W.3
-
29
-
-
0027534432
-
A human beta-globin gene fused to the human beta-globin locus control region is expressed at high levels in erythroid cells of mice engrafted with retrovirus-transduced hematopoietic stem cells
-
Plavec I, Papayannopoulou T, Maury C, Meyer F. A human beta-globin gene fused to the human beta-globin locus control region is expressed at high levels in erythroid cells of mice engrafted with retrovirus-transduced hematopoietic stem cells. Blood 1993; 81:1384-1392.
-
(1993)
Blood
, vol.81
, pp. 1384-1392
-
-
Plavec, I.1
Papayannopoulou, T.2
Maury, C.3
Meyer, F.4
-
30
-
-
0026519349
-
A 36-base-pair core sequence of locus control region enhances retrovirally transferred human beta-globin gene expression
-
USA
-
Chang JC, Liu D, Kan YW. A 36-base-pair core sequence of locus control region enhances retrovirally transferred human beta-globin gene expression. Proc Natl Acad Sci USA 1992; 89:3107-3110.
-
(1992)
Proc Natl Acad Sci
, vol.89
, pp. 3107-3110
-
-
Chang, J.C.1
Liu, D.2
Kan, Y.W.3
-
31
-
-
0025295890
-
High-level beta-globin expression after retroviral transfer of locus activation region-containing human beta-globin gene derivatives into murine erythroleukemia cells
-
USA
-
Novak U, Harris EA, Forrester W, et al. High-level beta-globin expression after retroviral transfer of locus activation region-containing human beta-globin gene derivatives into murine erythroleukemia cells. Proc Natl Acad Sci USA 1990; 87:3386-3390.
-
(1990)
Proc Natl Acad Sci
, vol.87
, pp. 3386-3390
-
-
Novak, U.1
Harris, E.A.2
Forrester, W.3
-
32
-
-
0032582826
-
Do LCRs open chromatin domains?
-
Higgs DR. Do LCRs open chromatin domains? Cell 1998; 95:299-302.
-
(1998)
Cell
, vol.95
, pp. 299-302
-
-
Higgs, D.R.1
-
33
-
-
0033215443
-
Looping versus linking: Toward a model for long-distance gene activation
-
Bulger M, Groudine M. Looping versus linking: toward a model for long-distance gene activation. Genes Dev 1999; 13:2465-2477.
-
(1999)
Genes Dev
, vol.13
, pp. 2465-2477
-
-
Bulger, M.1
Groudine, M.2
-
34
-
-
0032928813
-
Activation by locus control regions?
-
Grosveld F. Activation by locus control regions? Curr Opin Genet Dev 1999; 9:152-157.
-
(1999)
Curr Opin Genet Dev
, vol.9
, pp. 152-157
-
-
Grosveld, F.1
-
35
-
-
0034598936
-
Looping, linking, and chromatin activity: New insights into beta-globin locus regulation
-
Engel JD, Tanimoto K. Looping, linking, and chromatin activity: new insights into beta-globin locus regulation. Cell 2000; 100:499-502.
-
(2000)
Cell
, vol.100
, pp. 499-502
-
-
Engel, J.D.1
Tanimoto, K.2
-
36
-
-
0037090871
-
Activation of the beta-like globin genes in transgenic mice is dependent on the presence of the beta-locus control region
-
Navas PA, Li O, Peterson KR, et al. Activation of the beta-like globin genes in transgenic mice is dependent on the presence of the beta-locus control region. Hum Mol Genet 2002; 11:893-903.
-
(2002)
Hum Mol Genet
, vol.11
, pp. 893-903
-
-
Navas, P.A.1
Li, O.2
Peterson, K.R.3
-
37
-
-
0036297539
-
Stochastic patterns in globin gene expression are established prior to transcriptional activation and are clonally inherited
-
de Krom M, van de Corput M, von Lindern M, et al. Stochastic patterns in globin gene expression are established prior to transcriptional activation and are clonally inherited. Mol Cell 2002; 9:1319-1326.
-
(2002)
Mol Cell
, vol.9
, pp. 1319-1326
-
-
De Krom, M.1
Van De Corput, M.2
Von Lindern, M.3
-
38
-
-
0036532118
-
ChlPs of the beta-globin locus: Unraveling gene regulation within an active domain
-
Bulger M, Sawado T, Schubeler D, Groudine M. ChlPs of the beta-globin locus: unraveling gene regulation within an active domain. Curr Opin Genet Dev 2002; 12:170-177.
-
(2002)
Curr Opin Genet Dev
, vol.12
, pp. 170-177
-
-
Bulger, M.1
Sawado, T.2
Schubeler, D.3
Groudine, M.4
-
39
-
-
0036227306
-
The human beta-globin locus control region
-
Levings PP, Bungert J. The human beta-globin locus control region. Eur J Biochem 2002; 269:1589-1599.
-
(2002)
Eur J Biochem
, vol.269
, pp. 1589-1599
-
-
Levings, P.P.1
Bungert, J.2
-
40
-
-
1342302771
-
Globin gene transfer: A paradigm for transgene regulation and vector safety
-
Rivella S, Lisowski L, Sadelain M. Globin gene transfer: a paradigm for transgene regulation and vector safety. Gene Ther Reg 2003; 2:149-175.
-
(2003)
Gene Ther Reg
, vol.2
, pp. 149-175
-
-
Rivella, S.1
Lisowski, L.2
Sadelain, M.3
-
41
-
-
0030819839
-
Evaluation of beta-globin gene therapy constructs in single copy transgenic mice
-
Ellis J, Pasceri P, Tan-Un KC, et al. Evaluation of beta-globin gene therapy constructs in single copy transgenic mice. Nucleic Acids Res 1997; 25:1296-1302.
-
(1997)
Nucleic Acids Res
, vol.25
, pp. 1296-1302
-
-
Ellis, J.1
Pasceri, P.2
Tan-Un, K.C.3
-
42
-
-
0029984381
-
Production of genetically stable high-liter retroviral vectors that carry a human gamma-globin gene under the control of the alpha-globin locus control region
-
Ren S, Wong BY, Li J, et al. Production of genetically stable high-liter retroviral vectors that carry a human gamma-globin gene under the control of the alpha-globin locus control region. Blood 1996; 87:2518-2524.
-
(1996)
Blood
, vol.87
, pp. 2518-2524
-
-
Ren, S.1
Wong, B.Y.2
Li, J.3
-
43
-
-
0034472742
-
In vivo silencing of the human gamma-globin gene in murine erythroid cells following retroviral transduction
-
Lung HY, Meeus IS, Weinberg RS, Atweh GF. In vivo silencing of the human gamma-globin gene in murine erythroid cells following retroviral transduction. Blood Cells Mol Dis 2000; 26:613-619.
-
(2000)
Blood Cells Mol Dis
, vol.26
, pp. 613-619
-
-
Lung, H.Y.1
Meeus, I.S.2
Weinberg, R.S.3
Atweh, G.F.4
-
45
-
-
0034665986
-
A minimal ankyrin promoter linked to a human gamma-globin gene demonstrates erythroid specific copy number dependent expression with minimal position or enhancer dependence in transgenic mice
-
Sabatino DE, Wong C, Cline AP, et al. A minimal ankyrin promoter linked to a human gamma-globin gene demonstrates erythroid specific copy number dependent expression with minimal position or enhancer dependence in transgenic mice. J Biol Chem 2000; 275:28549-28554.
-
(2000)
J Biol Chem
, vol.275
, pp. 28549-28554
-
-
Sabatino, D.E.1
Wong, C.2
Cline, A.P.3
-
46
-
-
0034700114
-
Long-term expression of gamma-globin mRNA in mouse erythrocytes from retrovirus vectors containing the human gamma-globin gene fused to the ankyrin-1 promoter
-
USA
-
Sabatino DE, Seidel NE, Aviles-Mendoza GJ, et al. Long-term expression of gamma-globin mRNA in mouse erythrocytes from retrovirus vectors containing the human gamma-globin gene fused to the ankyrin-1 promoter. Proc Natl Acad Sci USA 2000; 97:13294-13299.
-
(2000)
Proc Natl Acad Sci
, vol.97
, pp. 13294-13299
-
-
Sabatino, D.E.1
Seidel, N.E.2
Aviles-Mendoza, G.J.3
-
47
-
-
0142214623
-
Persistent gamma-globin expression in adult transgenic mice is mediated by HPFH-2, HPFH-3, and HPFH-6 breakpoint sequences
-
Katsantoni EZ, Langeveld A, Wai AW. Persistent gamma-globin expression in adult transgenic mice is mediated by HPFH-2, HPFH-3, and HPFH-6 breakpoint sequences. Blood 2003; 102:3412-3419.
-
(2003)
Blood
, vol.102
, pp. 3412-3419
-
-
Katsantoni, E.Z.1
Langeveld, A.2
Wai, A.W.3
-
48
-
-
27544477087
-
Use of the hereditary persistence of fetal hemoglobin 2 enhancer to increase the expression of oncoretrovirus vectors for human gamma-globin
-
Fragkos M, Anagnou NP, Tubb J, Emery DW. Use of the hereditary persistence of fetal hemoglobin 2 enhancer to increase the expression of oncoretrovirus vectors for human gamma-globin. Gene Ther 2005; 12:1591-1600.
-
(2005)
Gene Ther
, vol.12
, pp. 1591-1600
-
-
Fragkos, M.1
Anagnou, N.P.2
Tubb, J.3
Emery, D.W.4
-
49
-
-
0343628721
-
Therapeutic haemoglobin synthesis in beta-thalassaemic mice expressing lentivirus-encoded human beta-globin
-
May C, Rivella S, Callegari J, et al. Therapeutic haemoglobin synthesis in beta-thalassaemic mice expressing lentivirus-encoded human beta-globin. Nature 2000; 406:82-86.
-
(2000)
Nature
, vol.406
, pp. 82-86
-
-
May, C.1
Rivella, S.2
Callegari, J.3
-
50
-
-
0036201352
-
Lentiviral vectors for the gene therapy of lymphohematological disorders
-
Salmon P, Trono D. Lentiviral vectors for the gene therapy of lymphohematological disorders. Curr Top Microbiol Immunol 2002; 261:211-227.
-
(2002)
Curr Top Microbiol Immunol
, vol.261
, pp. 211-227
-
-
Salmon, P.1
Trono, D.2
-
51
-
-
0036370361
-
Generation of HIV-1 derived lentiviral vectors
-
Follenzi A, Naldini L. Generation of HIV-1 derived lentiviral vectors. Meth Enzymol 2002; 346:454-465.
-
(2002)
Meth Enzymol
, vol.346
, pp. 454-465
-
-
Follenzi, A.1
Naldini, L.2
-
52
-
-
0028055281
-
Passage through mitosis is required for oncoretroviruses but not for the human immunodeficiency virus
-
Lewis PF, Emerman M. Passage through mitosis is required for oncoretroviruses but not for the human immunodeficiency virus. J Virol 1994; 68:510-516.
-
(1994)
J Virol
, vol.68
, pp. 510-516
-
-
Lewis, P.F.1
Emerman, M.2
-
53
-
-
0035524975
-
Intracellular trafficking of retroviral genomes during the early phase of infection: Viral exploitation of cellular pathways
-
Goff SP. Intracellular trafficking of retroviral genomes during the early phase of infection: viral exploitation of cellular pathways. J Gene Med 2001; 3:517-528.
-
(2001)
J Gene Med
, vol.3
, pp. 517-528
-
-
Goff, S.P.1
-
54
-
-
0031666744
-
Development of a self-inactivating lentivirus vector
-
Miyoshi H, Blomer U, Takahashi M, et al. Development of a self-inactivating lentivirus vector. J Virol 1998; 72:8150-8157.
-
(1998)
J Virol
, vol.72
, pp. 8150-8157
-
-
Miyoshi, H.1
Blomer, U.2
Takahashi, M.3
-
55
-
-
0033613827
-
Transduction of human CD34+ cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors
-
Miyoshi H, Smith KA, Mosier DE, et al. Transduction of human CD34+ cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors. Science 1999; 283:682-686.
-
(1999)
Science
, vol.283
, pp. 682-686
-
-
Miyoshi, H.1
Smith, K.A.2
Mosier, D.E.3
-
56
-
-
0034199171
-
Transduction of human CD34+ CD38- bone marrow and cord blood-derived SCID-repopulating cells with third-generation lentiviral vectors
-
Guenechea G, Gan OI, Inamitsu T, et al. Transduction of human CD34+ CD38- bone marrow and cord blood-derived SCID-repopulating cells with third-generation lentiviral vectors. MoI Ther 2000; 1:566-573.
-
(2000)
MoI Ther
, vol.1
, pp. 566-573
-
-
Guenechea, G.1
Gan, O.I.2
Inamitsu, T.3
-
57
-
-
0036194454
-
High levels of transduction of human dendritic cells with optimized SIV vectors
-
Mangeot PE, Duperrier K, Negre D, et al. High levels of transduction of human dendritic cells with optimized SIV vectors. Mol Ther 2002; 5:283-290.
-
(2002)
Mol Ther
, vol.5
, pp. 283-290
-
-
Mangeot, P.E.1
Duperrier, K.2
Negre, D.3
-
58
-
-
0036171327
-
Efficient gene delivery and targeted expression to hepatocytes in vivo by improved lentiviral vectors
-
Follenzi A, Sabatino G, Lombardo A, et al. Efficient gene delivery and targeted expression to hepatocytes in vivo by improved lentiviral vectors. Hum Gene Ther 2002; 13:243-260.
-
(2002)
Hum Gene Ther
, vol.13
, pp. 243-260
-
-
Follenzi, A.1
Sabatino, G.2
Lombardo, A.3
-
59
-
-
0035169569
-
Lentivirus-transduced human monocyte-derived dendritic cells efficiently stimulate antigen-specific cytotoxic T lymphocytes
-
Dyall J, Latouche JB, Schnell S, Sadelain M. Lentivirus-transduced human monocyte-derived dendritic cells efficiently stimulate antigen-specific cytotoxic T lymphocytes. Blood 2001; 97:114-121.
-
(2001)
Blood
, vol.97
, pp. 114-121
-
-
Dyall, J.1
Latouche, J.B.2
Schnell, S.3
Sadelain, M.4
-
60
-
-
0037114698
-
Lentiviral gene transfer and ex vivo expansion of human primitive stem cells capable of primary, secondary, and tertiary multilineage repopulation in NOD/SCID mice
-
Piacibello W, Bruno S, Sanavio F, et al. Lentiviral gene transfer and ex vivo expansion of human primitive stem cells capable of primary, secondary, and tertiary multilineage repopulation in NOD/SCID mice. Blood 2002; 100:4391-4400.
-
(2002)
Blood
, vol.100
, pp. 4391-4400
-
-
Piacibello, W.1
Bruno, S.2
Sanavio, F.3
-
61
-
-
0032956732
-
Transduction of human progenitor hematopoietic stem cells by human immunodeficiency virus type 1-based vectors is cell cycle dependent
-
Sutton RE, Reitsma MJ, Uchida N, Brown PO. Transduction of human progenitor hematopoietic stem cells by human immunodeficiency virus type 1-based vectors is cell cycle dependent. J Virol 1999; 73:3649-3660.
-
(1999)
J Virol
, vol.73
, pp. 3649-3660
-
-
Sutton, R.E.1
Reitsma, M.J.2
Uchida, N.3
Brown, P.O.4
-
62
-
-
0033789754
-
Issues in the manufacture and transplantation of genetically modified hematopoietic stem cells
-
Sadelain M, Frassoni F, Riviere I. Issues in the manufacture and transplantation of genetically modified hematopoietic stem cells. Curr Opin Hematol 2000; 7:364-377.
-
(2000)
Curr Opin Hematol
, vol.7
, pp. 364-377
-
-
Sadelain, M.1
Frassoni, F.2
Riviere, I.3
-
63
-
-
0034783643
-
Systematic determination of the packaging limit of lentiviral vectors
-
Kumar M, Keller B, Makalou N, Sutton RE. Systematic determination of the packaging limit of lentiviral vectors. Hum Gene Ther 2001; 12:1893-1905.
-
(2001)
Hum Gene Ther
, vol.12
, pp. 1893-1905
-
-
Kumar, M.1
Keller, B.2
Makalou, N.3
Sutton, R.E.4
-
64
-
-
0035861452
-
Correction of sickle cell disease in transgenic mouse models by gene therapy
-
Pawliuk R, Westerman KA, Fabry ME, et al. Correction of sickle cell disease in transgenic mouse models by gene therapy. Science 2001; 294:2368-2371.
-
(2001)
Science
, vol.294
, pp. 2368-2371
-
-
Pawliuk, R.1
Westerman, K.A.2
Fabry, M.E.3
-
66
-
-
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
-
Persons DA, Hargrove PW, Allay ER, et al. 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 2003; 101:2175-2183.
-
(2003)
Blood
, vol.101
, pp. 2175-2183
-
-
Persons, D.A.1
Hargrove, P.W.2
Allay, E.R.3
-
67
-
-
30544436003
-
A genetic strategy to treat sickle cell anemia by coregulating globin transgene expression and RNA interference
-
Samakoglu S, Lisowski L, Budak-Alpdogan T, et al. A genetic strategy to treat sickle cell anemia by coregulating globin transgene expression and RNA interference. Nat Biotechnol 2006; 24:89-94. This study supports the use of RNA interference to regulate gene expression in hematopoietic cells.
-
(2006)
Nat Biotechnol
, vol.24
, pp. 89-94
-
-
Samakoglu, S.1
Lisowski, L.2
Budak-Alpdogan, T.3
-
68
-
-
0036819745
-
Therapeutic globin gene delivery using lentiviral vectors
-
Rivella S, Sadelain M. Therapeutic globin gene delivery using lentiviral vectors. Curr Opin Mol Ther 2002; 4:505-514.
-
(2002)
Curr Opin Mol Ther
, vol.4
, pp. 505-514
-
-
Rivella, S.1
Sadelain, M.2
-
69
-
-
0037085774
-
Successful treatment of murine beta-thalassemia intermedia by transfer of the human beta-globin gene
-
May C, Rivella S, Chadburn A, Sadelain M. Successful treatment of murine beta-thalassemia intermedia by transfer of the human beta-globin gene. Blood 2002; 99:1902-1908.
-
(2002)
Blood
, vol.99
, pp. 1902-1908
-
-
May, C.1
Rivella, S.2
Chadburn, A.3
Sadelain, M.4
-
70
-
-
0037606048
-
A novel murine model of Cooley's anemia and its rescue by lentiviral mediated human β-globin gene transfer
-
Rivella S, May C, Chadburn A, et al. A novel murine model of Cooley's anemia and its rescue by lentiviral mediated human β-globin gene transfer. Blood 2003; 101:2932-2939.
-
(2003)
Blood
, vol.101
, pp. 2932-2939
-
-
Rivella, S.1
May, C.2
Chadburn, A.3
-
71
-
-
0028032847
-
Sickle cell disease of transgenic SAD mice
-
Trudel M, De Paepe ME, Chretien N, et al. Sickle cell disease of transgenic SAD mice. Blood 1994; 84:3189-3197.
-
(1994)
Blood
, vol.84
, pp. 3189-3197
-
-
Trudel, M.1
De Paepe, M.E.2
Chretien, N.3
-
72
-
-
1842408336
-
Transgenic knockout mice with exclusively human sickle hemoglobin and sickle cell disease
-
Paszty C, Brion CM, Manci E, et al. Transgenic knockout mice with exclusively human sickle hemoglobin and sickle cell disease. Science 1997; 278:876-878.
-
(1997)
Science
, vol.278
, pp. 876-878
-
-
Paszty, C.1
Brion, C.M.2
Manci, E.3
-
73
-
-
3042597832
-
A recombinant human hemoglobin with anti-sickling properties greater than fetal hemoglobin
-
Levasseur DN, Ryan TM, Reilly MP, et al. A recombinant human hemoglobin with anti-sickling properties greater than fetal hemoglobin. J Biol Chem 2004; 279:27518-27524.
-
(2004)
J Biol Chem
, vol.279
, pp. 27518-27524
-
-
Levasseur, D.N.1
Ryan, T.M.2
Reilly, M.P.3
-
74
-
-
1942454348
-
Anti-sickling effects of an endogenous human alpha-like globin
-
He S, Russell JE. Anti-sickling effects of an endogenous human alpha-like globin. Nat Med 2004; 10:365-367.
-
(2004)
Nat Med
, vol.10
, pp. 365-367
-
-
He, S.1
Russell, J.E.2
-
75
-
-
0033977775
-
Activation of human gamma-globin gene expression via triplex-forming oligonucleotide (TFO)-directed mutations in the gamma-globin gene 5′ flanking region
-
Xu XS, Glazer PM, Wang G. Activation of human gamma-globin gene expression via triplex-forming oligonucleotide (TFO)-directed mutations in the gamma-globin gene 5′ flanking region. Gene 2000; 242:219-228.
-
(2000)
Gene
, vol.242
, pp. 219-228
-
-
Xu, X.S.1
Glazer, P.M.2
Wang, G.3
-
76
-
-
0141743751
-
Efficient and specific repair of sickle β-globin RNA by trans-splicing ribozymes
-
Byun JH, Lan N, Long M, Sullenger BA, et al. Efficient and specific repair of sickle β-globin RNA by trans-splicing ribozymes. RNA 2003; 9:1254-1263.
-
(2003)
RNA
, vol.9
, pp. 1254-1263
-
-
Byun, J.H.1
Lan, N.2
Long, M.3
Sullenger, B.A.4
-
77
-
-
0035731827
-
Gene therapy for genetic haematological disorders and immunodeficiencies
-
Kohn DB. Gene therapy for genetic haematological disorders and immunodeficiencies. J Intern Med 2001; 249:379-390.
-
(2001)
J Intern Med
, vol.249
, pp. 379-390
-
-
Kohn, D.B.1
-
78
-
-
0001602245
-
Retroviral pathogenesis
-
Coffin AM, Hughes SH, Varmus HE, editors. New York: Cold Spring Harbor Laboratory Press
-
Rosenberg N, Jolicoeur P. Retroviral pathogenesis. In: Coffin AM, Hughes SH, Varmus HE, editors. Retroviruses. New York: Cold Spring Harbor Laboratory Press; 1997. pp. 475-585.
-
(1997)
Retroviruses
, pp. 475-585
-
-
Rosenberg, N.1
Jolicoeur, P.2
-
79
-
-
0036729878
-
High-throughput retroviral tagging to identify components of specific signaling pathways in cancer
-
Berns Mikkers H, Allen J, Knipscheer P, et al. High-throughput retroviral tagging to identify components of specific signaling pathways in cancer. Nat Genet 2002; 32:153-159.
-
(2002)
Nat Genet
, vol.32
, pp. 153-159
-
-
Berns Mikkers, H.1
Allen, J.2
Knipscheer, P.3
-
80
-
-
0036725051
-
New genes involved in cancer identified by retroviral tagging
-
Suzuki T, Shen H, Akagi K, et al. New genes involved in cancer identified by retroviral tagging. Nat Genet 2002; 32:166-174.
-
(2002)
Nat Genet
, vol.32
, pp. 166-174
-
-
Suzuki, T.1
Shen, H.2
Akagi, K.3
-
81
-
-
0037443457
-
Side effects of retroviral gene transfer into hematopoietic stem cells
-
Baum C, Dullmann J, Li Z, et al. Side effects of retroviral gene transfer into hematopoietic stem cells. Blood 2003; 101:2099-2114.
-
(2003)
Blood
, vol.101
, pp. 2099-2114
-
-
Baum, C.1
Dullmann, J.2
Li, Z.3
-
82
-
-
10744233858
-
American Society of Gene Therapy (ASGT) ad hoc subcommittee on retroviral-mediated gene transfer to hematopoietic stem cells
-
Kohn DB, Sadelain M, Dunbar C, et al. American Society of Gene Therapy (ASGT) ad hoc subcommittee on retroviral-mediated gene transfer to hematopoietic stem cells. Mol Ther 2003; 8:180-187.
-
(2003)
Mol Ther
, vol.8
, pp. 180-187
-
-
Kohn, D.B.1
Sadelain, M.2
Dunbar, C.3
-
83
-
-
0037134031
-
Murine leukemia induced by retroviral gene marking
-
Li Z, Dullmann J, Schiedlmeier B, et al. Murine leukemia induced by retroviral gene marking. Science 2002; 296:497.
-
(2002)
Science
, vol.296
, pp. 497
-
-
Li, Z.1
Dullmann, J.2
Schiedlmeier, B.3
-
84
-
-
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; 302:415-419.
-
(2003)
Science
, vol.302
, pp. 415-419
-
-
Hacein-Bey-Abina, S.1
Von Kalle, C.2
Schmidt, M.3
-
85
-
-
0042738935
-
Occurrence of leukaemia following gene therapy of X-linked SCID
-
Kohn DB, Sadelain M, Glorioso JC. Occurrence of leukaemia following gene therapy of X-linked SCID. Nat Rev Cancer 2003; 3:477-488.
-
(2003)
Nat Rev Cancer
, vol.3
, pp. 477-488
-
-
Kohn, D.B.1
Sadelain, M.2
Glorioso, J.C.3
-
86
-
-
0347634456
-
Gene therapy insertional mutagenesis insights
-
Dave UP, Jenkins NA, Copeland NG. Gene therapy insertional mutagenesis insights. Science 2004; 303:333.
-
(2004)
Science
, vol.303
, pp. 333
-
-
Dave, U.P.1
Jenkins, N.A.2
Copeland, N.G.3
-
87
-
-
1842533449
-
Insertional oncogenesis in human gene therapy: How much of a risk?
-
Sadelain M. Insertional oncogenesis in human gene therapy: how much of a risk? Gene Ther 2004; 11:567-573.
-
(2004)
Gene Ther
, vol.11
, pp. 567-573
-
-
Sadelain, M.1
-
88
-
-
0034000919
-
The cHS4 insulator increases the probability of retroviral expression at random chromosomal integration sites
-
Rivella S, Callegari JA, May C, et al. The cHS4 insulator increases the probability of retroviral expression at random chromosomal integration sites. J Virol 2000; 74:4679-4687.
-
(2000)
J Virol
, vol.74
, pp. 4679-4687
-
-
Rivella, S.1
Callegari, J.A.2
May, C.3
-
89
-
-
0034255145
-
A chromatin insulator protects retrovirus vectors from chromosomal position effects
-
USA
-
Emery DW, Yannaki E, Tubb J, Stamatoyannopoulos G. A chromatin insulator protects retrovirus vectors from chromosomal position effects. Proc Natl Acad Sci USA 2000; 97:9150-9155.
-
(2000)
Proc Natl Acad Sci
, vol.97
, pp. 9150-9155
-
-
Emery, D.W.1
Yannaki, E.2
Tubb, J.3
Stamatoyannopoulos, G.4
-
90
-
-
0036099418
-
Topological constraints governing the use of the chicken HS4 chromatin insulator in oncoretrovirus vectors
-
Yannaki E, Tubb J, Aker M, et al. Topological constraints governing the use of the chicken HS4 chromatin insulator in oncoretrovirus vectors. Mol Ther 2002; 5:589-598.
-
(2002)
Mol Ther
, vol.5
, pp. 589-598
-
-
Yannaki, E.1
Tubb, J.2
Aker, M.3
-
91
-
-
0037105522
-
Development of virus vectors for gene therapy of beta chain hemoglobinopathies: Flanking with a chromatin insulator reduces gamma-globin gene silencing in vivo
-
Emery DW, Yannaki E, Tubb J, et al. Development of virus vectors for gene therapy of beta chain hemoglobinopathies: flanking with a chromatin insulator reduces gamma-globin gene silencing in vivo. Blood 2002; 100:2012-2019.
-
(2002)
Blood
, vol.100
, pp. 2012-2019
-
-
Emery, D.W.1
Yannaki, E.2
Tubb, J.3
-
92
-
-
32244434403
-
Correction of the sickle cell mutation in embryonic stem cells
-
USA
-
Chang JC, Ye L, Kan YW. Correction of the sickle cell mutation in embryonic stem cells. Proc Natl Acad Sci USA 2006; 103:1036-1040. The embryonic stem cell technology suggests that a therapeutic cloning approach may be feasible for treatment of sickle cell anemia.
-
(2006)
Proc Natl Acad Sci
, vol.103
, pp. 1036-1040
-
-
Chang, J.C.1
Ye, L.2
Kan, Y.W.3
|