-
1
-
-
0034724857
-
Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease
-
+ cells from two infants with X-linked SCID led to immune restoration
-
+ cells from two infants with X-linked SCID led to immune restoration.
-
(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
Nusbaum, P.6
Selz, F.7
Hue, C.8
Certain, S.9
Casanova, J.L.10
-
2
-
-
0037129435
-
Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy
-
This paper reported results of longer follow-up and more subjects from the study described in [1•] with the therapeutic results persisting as long as three years for the first subjects
-
Hacein-Bey-Abina S., Le Deist F., Carlier F., Bouneaud C., Hue C., De Villartay J.P., Thrasher A.J., Wulffraat N., Sorensen R., Dupuis-Girod S., et al. Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. N Engl J Med. 346:2002;1185-1193. This paper reported results of longer follow-up and more subjects from the study described in [1•] with the therapeutic results persisting as long as three years for the first subjects.
-
(2002)
N Engl J Med
, vol.346
, pp. 1185-1193
-
-
Hacein-Bey-Abina, S.1
Le Deist, F.2
Carlier, F.3
Bouneaud, C.4
Hue, C.5
De Villartay, J.P.6
Thrasher, A.J.7
Wulffraat, N.8
Sorensen, R.9
Dupuis-Girod, S.10
-
3
-
-
0037189401
-
Correction of ADA-SCID by stem cell gene therapy combined with non-myeloablative conditioning
-
+ cells. Novel aspects from previous studies included withholding administration of PEG-ADA enzyme replacement therapy to preserve a selective advantage of ADA gene-corrected T cells and administration of low dosages of chemotherapy to partially ablate the recipient's bone marrow
-
+ cells. Novel aspects from previous studies included withholding administration of PEG-ADA enzyme replacement therapy to preserve a selective advantage of ADA gene-corrected T cells and administration of low dosages of chemotherapy to partially ablate the recipient's bone marrow.
-
(2002)
Science
, vol.296
, pp. 2410-2413
-
-
Aiuti, A.1
Slavin, S.2
Aker, M.3
Ficara, F.4
Deola, S.5
Mortellaro, A.6
Morecki, S.7
Andolfi, G.8
Tabucchi, A.9
Carlucci, F.10
-
4
-
-
0032578385
-
HIV, but not murine leukemia virus, vectors mediate high efficiency gene transfer into freshly isolated G0/G1 human hematopoietic stem cells
-
Uchida N., Sutton R.E., Friera A.M., He D., Reisma M.J., Chang W.C., Veres G., Scollay R., Weissman I.L. HIV, but not murine leukemia virus, vectors mediate high efficiency gene transfer into freshly isolated G0/G1 human hematopoietic stem cells. Proc Natl Acad Sci USA. 95:1998;11939-11944.
-
(1998)
Proc Natl Acad Sci USA
, vol.95
, pp. 11939-11944
-
-
Uchida, N.1
Sutton, R.E.2
Friera, A.M.3
He, D.4
Reisma, M.J.5
Chang, W.C.6
Veres, G.7
Scollay, R.8
Weissman, I.L.9
-
6
-
-
13044309472
-
+CD38- human hematopoietic cells by HIV-1-based lentiviral vectors
-
+CD38- human hematopoietic cells by HIV-1-based lentiviral vectors. Proc Natl Acad Sci USA. 96:1999;2988-2993.
-
(1999)
Proc Natl Acad Sci USA
, 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
-
8
-
-
0027376309
-
A nuclear localization signal within HIV-1 matrix protein that governs infection of non-dividing cells
-
Bukrinsky M.I., Haggerty S., Dempsy M.P., Sharova N., Adzhubel A., Spitz L., Lewis P., Goldfarb D., Emerman M., Stevenson M. A nuclear localization signal within HIV-1 matrix protein that governs infection of non-dividing cells. Nature. 365:1993;666-669.
-
(1993)
Nature
, vol.365
, pp. 666-669
-
-
Bukrinsky, M.I.1
Haggerty, S.2
Dempsy, M.P.3
Sharova, N.4
Adzhubel, A.5
Spitz, L.6
Lewis, P.7
Goldfarb, D.8
Emerman, M.9
Stevenson, M.10
-
9
-
-
0030987672
-
HIV-1 infection of nondividing cells through recognition of integrase by the importin/karyopherin pathway
-
Gallay P., Hope T., Chin D., Trono D. HIV-1 infection of nondividing cells through recognition of integrase by the importin/karyopherin pathway. Proc Natl Acad Sci USA. 94:1997;9825-9830.
-
(1997)
Proc Natl Acad Sci USA
, vol.94
, pp. 9825-9830
-
-
Gallay, P.1
Hope, T.2
Chin, D.3
Trono, D.4
-
10
-
-
0035798131
-
Dynamic disruptions in nuclear envelope architecture and integrity induced by HIV-1 Vpr
-
De Noronha C.M.C., Sherman M.P., Lin H.W., Cavrois M.V., Moir R.D., Goldman R.D., Greene W.C. Dynamic disruptions in nuclear envelope architecture and integrity induced by HIV-1 Vpr. Science. 294:2001;1105-1108.
-
(2001)
Science
, vol.294
, pp. 1105-1108
-
-
De Noronha, C.M.C.1
Sherman, M.P.2
Lin, H.W.3
Cavrois, M.V.4
Moir, R.D.5
Goldman, R.D.6
Greene, W.C.7
-
11
-
-
0034646513
-
HIV-1 genome nuclear import is mediated by a central DNA flap
-
Detailed analysis of cPPT function, demonstrating accumulation of cPPT-defective PICs near the cytoplasmic surface of the nuclear membrane, whereas PICs containing the wild-type cPPT trafficked efficiently into the nucleus
-
Zennou V., Petit C., Guetard D., Nerhbass U., Montagnier L., Charneau P. HIV-1 genome nuclear import is mediated by a central DNA flap. Cell. 101:2000;173-185. Detailed analysis of cPPT function, demonstrating accumulation of cPPT-defective PICs near the cytoplasmic surface of the nuclear membrane, whereas PICs containing the wild-type cPPT trafficked efficiently into the nucleus.
-
(2000)
Cell
, vol.101
, pp. 173-185
-
-
Zennou, V.1
Petit, C.2
Guetard, D.3
Nerhbass, U.4
Montagnier, L.5
Charneau, P.6
-
12
-
-
0034040751
-
Gene transfer by lentiviral vectors limited by nuclear translocation and rescued by HIV-1 pol sequences
-
Follenzi A., Ailles L.E., Bakovic S., Geuna M., Naldini L. Gene transfer by lentiviral vectors limited by nuclear translocation and rescued by HIV-1 pol sequences. Nat Genet. 25:2000;217-222.
-
(2000)
Nat Genet
, vol.25
, pp. 217-222
-
-
Follenzi, A.1
Ailles, L.E.2
Bakovic, S.3
Geuna, M.4
Naldini, L.5
-
13
-
-
0034672356
-
The human immunodeficiency virus type-1 central DNA flap is a crucial determinant for lentiviral vector nuclear import and gene transduction of human hematopoietic stem cells
-
Sirven A., Pflumio F., Zennou V., Titeux M., Vainchenker W., Coulomel L., Dubart-Kupperscmitt A., Charneau P. The human immunodeficiency virus type-1 central DNA flap is a crucial determinant for lentiviral vector nuclear import and gene transduction of human hematopoietic stem cells. Blood. 96:2000;4103-4110.
-
(2000)
Blood
, vol.96
, pp. 4103-4110
-
-
Sirven, A.1
Pflumio, F.2
Zennou, V.3
Titeux, M.4
Vainchenker, W.5
Coulomel, L.6
Dubart-Kupperscmitt, A.7
Charneau, P.8
-
15
-
-
0033843219
-
Improved titers of HIV-based lentiviral vectors using the SRV-1 constitutive transport element
-
Mautino M.R., Keiser N., Morgan R.A. Improved titers of HIV-based lentiviral vectors using the SRV-1 constitutive transport element. Gene Ther. 7:2000;1421-1424.
-
(2000)
Gene Ther
, vol.7
, pp. 1421-1424
-
-
Mautino, M.R.1
Keiser, N.2
Morgan, R.A.3
-
16
-
-
0034849315
-
Modified HIV-1 based lentiviral vectors have an effect on viral transduction efficiency and gene expression in vitro and in vivo
-
Park F., Kay M. Modified HIV-1 based lentiviral vectors have an effect on viral transduction efficiency and gene expression in vitro and in vivo. Mol Ther. 4:2001;164-173.
-
(2001)
Mol Ther
, vol.4
, pp. 164-173
-
-
Park, F.1
Kay, M.2
-
17
-
-
0032519764
-
+CD38-human hematopoietic progenitors assessed in immune-deficient mice
-
+CD38-human hematopoietic progenitors assessed in immune-deficient mice. Blood. 91:1998;1243-1255.
-
(1998)
Blood
, vol.91
, pp. 1243-1255
-
-
Dao, M.A.1
Shah, A.J.2
Crooks, G.M.3
Nolta, J.A.4
-
18
-
-
0031019451
-
+ progenitors to sustain long-term hematopoiesis in immune-deficient mice after ex vivo retroviral-mediated transduction
-
+ progenitors to sustain long-term hematopoiesis in immune-deficient mice after ex vivo retroviral-mediated transduction. Blood. 89:1997;446-456.
-
(1997)
Blood
, vol.89
, pp. 446-456
-
-
Dao, M.A.1
Hannum, C.H.2
Kohn, D.B.3
Nolta, J.A.4
-
19
-
-
0033964863
-
+CD38- cells in ex vivo culture during retroviral transduction without a corresponding increase in SCID repopulating cell (SRC) frequency: Dissociation of SRC phenotype repopulating cell (SRC) frequency: Dissociation of SRC phenotype and function
-
+CD38- cells in ex vivo culture during retroviral transduction without a corresponding increase in SCID repopulating cell (SRC) frequency: dissociation of SRC phenotype repopulating cell (SRC) frequency: dissociation of SRC phenotype and function. Blood. 95:2000;102-110.
-
(2000)
Blood
, vol.95
, pp. 102-110
-
-
Dorrell, C.1
Gan, O.I.2
Pereira, D.S.3
Hawley, R.G.4
Dick, J.E.5
-
20
-
-
0034199171
-
+ CD38- bone marrow and cord blood-derived SCID-repopulating cells with third-generation lentiviral vectors
-
+ CD38- bone marrow and cord blood-derived SCID-repopulating cells with third-generation lentiviral vectors. Mol Ther. 1:2000;566-573.
-
(2000)
Mol Ther
, vol.1
, pp. 566-573
-
-
Guenechea, G.1
Gan, O.I.2
Inamitsu, T.3
Dorrell, C.4
Pereira, D.S.5
Kelly, M.6
Naldini, L.7
Dick, J.E.8
-
21
-
-
0032956732
-
Transduction of human progenitor hematopoietic stem cells by human immunodeficiency virus type 1-based vectors is cell-cycle dependent
-
Sutton R.E., Reitsma M.J., Uchida N., Brown P.O. Transduction of human progenitor hematopoietic stem cells by human immunodeficiency virus type 1-based vectors is cell-cycle dependent. J Virol. 73:1999;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
-
22
-
-
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
-
Korin Y.D., Zack J.A. 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
-
23
-
-
0032535694
-
Adhesion to fibronectin maintains regenerative capacity during ex vivo culture and transduction of human hematopoietic stem and progenitor cells
-
Dao M.A., Hashino K., Kato I., Nolta J.A. Adhesion to fibronectin maintains regenerative capacity during ex vivo culture and transduction of human hematopoietic stem and progenitor cells. Blood. 92:1998;4612-4621.
-
(1998)
Blood
, vol.92
, pp. 4612-4621
-
-
Dao, M.A.1
Hashino, K.2
Kato, I.3
Nolta, J.A.4
-
25
-
-
0034669932
-
High-level transgene expression in human hematopoietic progenitors and differentiated blood lineages after transduction with improved lentiviral vectors
-
Salmon P., Kindler V., Ducrey O., Chapuis B., Zubler R.H., Trono D. High-level transgene expression in human hematopoietic progenitors and differentiated blood lineages after transduction with improved lentiviral vectors. Blood. 96:2000;3392-3398.
-
(2000)
Blood
, vol.96
, pp. 3392-3398
-
-
Salmon, P.1
Kindler, V.2
Ducrey, O.3
Chapuis, B.4
Zubler, R.H.5
Trono, D.6
-
26
-
-
0031710033
-
A third-generation lentivirus vector with a conditional packaging system
-
Dull T., Zufferey R., Kelly M., Mandel R.J., Nguyen M., Trono D., Naldini L. A third-generation lentivirus vector with a conditional packaging system. J Virol. 72:1998;8463-8471.
-
(1998)
J Virol
, vol.72
, pp. 8463-8471
-
-
Dull, T.1
Zufferey, R.2
Kelly, M.3
Mandel, R.J.4
Nguyen, M.5
Trono, D.6
Naldini, L.7
-
27
-
-
0031743608
-
Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery
-
Zufferey R., Dull T., Mandel R.J., Bukovsky A., Quiroz D., Naldini L., Trono D. 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
-
28
-
-
0034694022
-
Lentivirus vector mobilization and spread by human immunodeficiency virus
-
Evans J., Garcia J.V. Lentivirus vector mobilization and spread by human immunodeficiency virus. Hum Gene Ther. 11:2000;2331-2339.
-
(2000)
Hum Gene Ther
, vol.11
, pp. 2331-2339
-
-
Evans, J.1
Garcia, J.V.2
-
29
-
-
0032796643
-
Interaction of human immunodeficiency virus-derived vectors with wild-type virus in transduced cells
-
Bukovsky A.A., Song J., Naldini L. Interaction of human immunodeficiency virus-derived vectors with wild-type virus in transduced cells. J Virol. 73:1999;7087-7092.
-
(1999)
J Virol
, vol.73
, pp. 7087-7092
-
-
Bukovsky, A.A.1
Song, J.2
Naldini, L.3
-
30
-
-
0033566372
-
Self-inactivating lentiviral vectors with U3 and U5 modifications
-
Iwakuma T., Cui Y., Chang L.J. Self-inactivating lentiviral vectors with U3 and U5 modifications. Virology. 261:1999;120-132.
-
(1999)
Virology
, vol.261
, pp. 120-132
-
-
Iwakuma, T.1
Cui, Y.2
Chang, L.J.3
-
31
-
-
0034988922
-
Generation of a stable cell line producing high-titer self-inactivating lentiviral vectors
-
Xu K., Ma H., McCown T.J., Verma I.M., Kafri T. Generation of a stable cell line producing high-titer self-inactivating lentiviral vectors. Mol Ther. 3:2001;97-104.
-
(2001)
Mol Ther
, vol.3
, pp. 97-104
-
-
Xu, K.1
Ma, H.2
McCown, T.J.3
Verma, I.M.4
Kafri, T.5
-
32
-
-
0033059216
-
Contributions of viral splice sites and cis-regulatory elements to lentivirus vector function
-
This is the first paper describing the generation of an HIV-1-based vector containing a minimum amount of residual HIV-1 genome
-
Cui Y., Iwakuma T., Chang L.J. Contributions of viral splice sites and cis-regulatory elements to lentivirus vector function. J Virol. 73:1999;6171-6176. This is the first paper describing the generation of an HIV-1-based vector containing a minimum amount of residual HIV-1 genome.
-
(1999)
J Virol
, vol.73
, pp. 6171-6176
-
-
Cui, Y.1
Iwakuma, T.2
Chang, L.J.3
-
33
-
-
0034218268
-
Development of a novel trans-lentiviral vector that affords predictable safety
-
To eliminate the generation of recombinants, the authors split gag/gag-pol into two plasmids: one which expresses Gag polyprotein and protease, and another expressing reverse transcriptase and integrase fused to Vpr to ensure their inclusion in vector particles
-
Wu X., Wakefield J.K., Liu H., Xiao H., Kralovics R., Prchal J.R., Kappes J.C. Development of a novel trans-lentiviral vector that affords predictable safety. Mol Ther. 2:2000;47-55. To eliminate the generation of recombinants, the authors split gag/gag-pol into two plasmids: one which expresses Gag polyprotein and protease, and another expressing reverse transcriptase and integrase fused to Vpr to ensure their inclusion in vector particles.
-
(2000)
Mol Ther
, vol.2
, pp. 47-55
-
-
Wu, X.1
Wakefield, J.K.2
Liu, H.3
Xiao, H.4
Kralovics, R.5
Prchal, J.R.6
Kappes, J.C.7
-
34
-
-
0034914353
-
A new-generation stable inducible packaging cell line for lentiviral vectors
-
This paper reveals the challenges of generating a lentiviral packaging cell line, in that packaging cell clones capable of producing high titer vector are rare, and that the infectivity of the vector particles produced by different clones varies widely
-
Farson D., Witt R., McGuinness R., Dull T., Kelly M., Song J., Radeke R., Bukovsky A., Consiglio A., Naldini L. A new-generation stable inducible packaging cell line for lentiviral vectors. Hum Gene Ther. 12:2001;981-987. This paper reveals the challenges of generating a lentiviral packaging cell line, in that packaging cell clones capable of producing high titer vector are rare, and that the infectivity of the vector particles produced by different clones varies widely.
-
(2001)
Hum Gene Ther
, vol.12
, pp. 981-987
-
-
Farson, D.1
Witt, R.2
McGuinness, R.3
Dull, T.4
Kelly, M.5
Song, J.6
Radeke, R.7
Bukovsky, A.8
Consiglio, A.9
Naldini, L.10
-
35
-
-
0029996147
-
In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector
-
Naldini L., Blomer U., Gallay P., Ory D., Mulligan R., Gage F.H., Verma I.M., Trono D. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science. 272:1996;263-267.
-
(1996)
Science
, vol.272
, pp. 263-267
-
-
Naldini, L.1
Blomer, U.2
Gallay, P.3
Ory, D.4
Mulligan, R.5
Gage, F.H.6
Verma, I.M.7
Trono, D.8
-
36
-
-
0034322159
-
Lentiviral vectors for enhanced gene expression in human hematopoietic cells
-
Ramezani A., Hawley T.S., Hawley R.G. Lentiviral vectors for enhanced gene expression in human hematopoietic cells. Mol Ther. 2:2000;458-469.
-
(2000)
Mol Ther
, vol.2
, pp. 458-469
-
-
Ramezani, A.1
Hawley, T.S.2
Hawley, R.G.3
-
37
-
-
85031364232
-
Optimization of HIV-1-based lentiviral vectors for gene-modification of leukemia cells
-
Logan A.C., Kohn D.B. Optimization of HIV-1-based lentiviral vectors for gene-modification of leukemia cells. Mol Ther, 5:2000;s426.
-
(2000)
Mol Ther
, vol.5
-
-
Logan, A.C.1
Kohn, D.B.2
-
38
-
-
0034071645
-
A murine leukemia virus (MuLV) long terminal repeat derived from Rhesus macaques in the context of a lentivirus vector and MuLV gag sequence results in high-level gene expression in human T lymphocytes
-
Kung S.K., An D.S., Chen I.S. A murine leukemia virus (MuLV) long terminal repeat derived from Rhesus macaques in the context of a lentivirus vector and MuLV gag sequence results in high-level gene expression in human T lymphocytes. J Virol. 74:2000;3668-3681.
-
(2000)
J Virol
, vol.74
, pp. 3668-3681
-
-
Kung, S.K.1
An, D.S.2
Chen, I.S.3
-
39
-
-
0036252492
-
High-level transduction and gene expression in hematopoietic repopulating cells using a human immunodeficiency virus type 1-based lentiviral vector containing an internal spleen focus forming virus promoter
-
Demaison C., Parsley K., Brouns G., Scherr M., Battmer K., Kinnon C., Grez M., Thrasher A.J. High-level transduction and gene expression in hematopoietic repopulating cells using a human immunodeficiency virus type 1-based lentiviral vector containing an internal spleen focus forming virus promoter. Hum Gene Ther. 13:2000;803-813.
-
(2000)
Hum Gene Ther
, vol.13
, pp. 803-813
-
-
Demaison, C.1
Parsley, K.2
Brouns, G.3
Scherr, M.4
Battmer, K.5
Kinnon, C.6
Grez, M.7
Thrasher, A.J.8
-
41
-
-
0034994357
-
High levels of transgene expression following transduction of long-term NOD/SCID-repopulating human cells with a modified lentiviral vector
-
Gao Z., Golob J., Tanavde V.M., Civin C.I., Hawley R.G., Cheng L. High levels of transgene expression following transduction of long-term NOD/SCID-repopulating human cells with a modified lentiviral vector. Stem Cells. 19:2001;247-259.
-
(2001)
Stem Cells
, vol.19
, pp. 247-259
-
-
Gao, Z.1
Golob, J.2
Tanavde, V.M.3
Civin, C.I.4
Hawley, R.G.5
Cheng, L.6
-
42
-
-
0033994632
-
Expression of human Wiskott-Aldrich syndrome protein in patients' cells leads to partial correction of a phenotypic abnormality of cell surface glycoproteins
-
Huang M.M., Wong A., Tsuboi S., Yu X.J., Oh-Eda M., Derry J.M., Franke U., Fukuda M., Weinberg K.I., Kohn D.B. Expression of human Wiskott-Aldrich syndrome protein in patients' cells leads to partial correction of a phenotypic abnormality of cell surface glycoproteins. Gene Ther. 7:2000;314-320.
-
(2000)
Gene Ther
, vol.7
, pp. 314-320
-
-
Huang, M.M.1
Wong, A.2
Tsuboi, S.3
Yu, X.J.4
Oh-Eda, M.5
Derry, J.M.6
Franke, U.7
Fukuda, M.8
Weinberg, K.I.9
Kohn, D.B.10
-
44
-
-
0036193248
-
Robust and efficient regulation of transgene expression in vivo by improved tetracycline-dependent lentiviral vectors
-
Vigna E., Cavalieri S., Ailles L., Geuna M., Loew R., Bujard H., Naldini L. Robust and efficient regulation of transgene expression in vivo by improved tetracycline-dependent lentiviral vectors. Mol Ther. 5:2002;252-261.
-
(2002)
Mol Ther
, vol.5
, pp. 252-261
-
-
Vigna, E.1
Cavalieri, S.2
Ailles, L.3
Geuna, M.4
Loew, R.5
Bujard, H.6
Naldini, L.7
-
45
-
-
0343628721
-
Therapeutic haemoglobin synthesis in β-thalassaemic mice expressing lentivirus-encoded human β-globin
-
This paper represents a giant step toward effective gene therapy for hemoglobinopathies. Lentiviral vectors carrying sequences of the β-globin LCR expressed at physiologically relevant levels in erythrocytes of mice after transduction of HSCs
-
May C., Rivella S., Callegari J., Heller G., Gaensler K.M., Luzzatto L., Sadelain M. Therapeutic haemoglobin synthesis in β-thalassaemic mice expressing lentivirus-encoded human β-globin. Nature. 406:2000;82-86. This paper represents a giant step toward effective gene therapy for hemoglobinopathies. Lentiviral vectors carrying sequences of the β-globin LCR expressed at physiologically relevant levels in erythrocytes of mice after transduction of HSCs.
-
(2000)
Nature
, vol.406
, pp. 82-86
-
-
May, C.1
Rivella, S.2
Callegari, J.3
Heller, G.4
Gaensler, K.M.5
Luzzatto, L.6
Sadelain, M.7
-
46
-
-
0037085774
-
Successful treatment of murine β-thalassemia intermediates by transfer of the human β-globin gene
-
May C., Rivella S., Chadburn A., Sadelain M. Successful treatment of murine β-thalassemia intermediates by transfer of the human β-globin gene. Blood. 99:2002;1902-1908.
-
(2002)
Blood
, vol.99
, pp. 1902-1908
-
-
May, C.1
Rivella, S.2
Chadburn, A.3
Sadelain, M.4
-
47
-
-
0035861452
-
Correction of sickle cell disease in transgenic mouse models by gene therapy
-
Pawliuk R., Westerman K.A., Fabry M.E., Payen E., Tighe R., Bouhassira E.E., Acharya S.A., Ellis J., London I.M., Eaves C.J., et al. Correction of sickle cell disease in transgenic mouse models by gene therapy. Science. 294:2001;2268.
-
(2001)
Science
, vol.294
, pp. 2268
-
-
Pawliuk, R.1
Westerman, K.A.2
Fabry, M.E.3
Payen, E.4
Tighe, R.5
Bouhassira, E.E.6
Acharya, S.A.7
Ellis, J.8
London, I.M.9
Eaves, C.J.10
-
48
-
-
0035525735
-
High-level erythroid-specific gene expression in primary human and murine hematopoietic cells with self-inactivating lentiviral vectors
-
Moreau-Gaudry F., Xia P., Jiang G., Perelman N.P., Bauer G., Ellis J., Surinya K.H., Mavilio F., Shen C.K., Malik P. High-level erythroid-specific gene expression in primary human and murine hematopoietic cells with self-inactivating lentiviral vectors. Blood. 98:2001;2664-2672.
-
(2001)
Blood
, vol.98
, pp. 2664-2672
-
-
Moreau-Gaudry, F.1
Xia, P.2
Jiang, G.3
Perelman, N.P.4
Bauer, G.5
Ellis, J.6
Surinya, K.H.7
Mavilio, F.8
Shen, C.K.9
Malik, P.10
-
49
-
-
0035037078
-
Locus control region of the human CD2 gene in a lentivirus vector confers position-independent transgene expression
-
Kowolik C.M., Hu J., Yee J.K. Locus control region of the human CD2 gene in a lentivirus vector confers position-independent transgene expression. J Virol. 75:2001;4641-4648.
-
(2001)
J Virol
, vol.75
, pp. 4641-4648
-
-
Kowolik, C.M.1
Hu, J.2
Yee, J.K.3
-
50
-
-
0011559071
-
Regulated transgene expression in human and murine B-lymphoid cells from lentiviral vectors carrying the immunoglobulin heavy chain enhancer and MARs
-
Lutzko C., Peterson D., Senadheera D., Kohn D.B. Regulated transgene expression in human and murine B-lymphoid cells from lentiviral vectors carrying the immunoglobulin heavy chain enhancer and MARs. Mol Ther. 5:2002;s425.
-
(2002)
Mol Ther
, vol.5
, pp. 425
-
-
Lutzko, C.1
Peterson, D.2
Senadheera, D.3
Kohn, D.B.4
-
51
-
-
0037079725
-
Targeting transgene expression to antigen-presenting cells derived from lentivirus-transduced engrafting human hematopoietic stem/progenitor cells
-
Cui Y., Golob J., Kelleher E., Ye Z., Pardoll D., Cheng L. Targeting transgene expression to antigen-presenting cells derived from lentivirus-transduced engrafting human hematopoietic stem/progenitor cells. Blood. 99:2002;399-408.
-
(2002)
Blood
, vol.99
, pp. 399-408
-
-
Cui, Y.1
Golob, J.2
Kelleher, E.3
Ye, Z.4
Pardoll, D.5
Cheng, L.6
-
52
-
-
85031348389
-
Lentiviral vectors utilizing the chicken β-globin 5′HS4 insulator and the human interferon-β scaffold attachment region for high level and sustained factor VIII gene expression
-
Ramezani A., Hawley T.S., Hawley R.G. Lentiviral vectors utilizing the chicken β-globin 5′HS4 insulator and the human interferon-β scaffold attachment region for high level and sustained factor VIII gene expression. Mol Ther. 5:2002;s425.
-
(2002)
Mol Ther
, vol.5
, pp. 425
-
-
Ramezani, A.1
Hawley, T.S.2
Hawley, R.G.3
-
53
-
-
0034783643
-
Systematic determination of the packaging limit of lentiviral vectors
-
In a highly systematic manner, the effects of transgene insert size on vector performance were determined; titers were inversely related to insert size
-
Kumar M., Keller B., Makalou N., Sutton R.E. Systematic determination of the packaging limit of lentiviral vectors. Hum Gene Ther. 12:2001;1893-1905. In a highly systematic manner, the effects of transgene insert size on vector performance were determined; titers were inversely related to insert size.
-
(2001)
Hum Gene Ther
, vol.12
, pp. 1893-1905
-
-
Kumar, M.1
Keller, B.2
Makalou, N.3
Sutton, R.E.4
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