-
1
-
-
0035793325
-
Gene transfer as an approach to treating hemophilia
-
High KA. Gene transfer as an approach to treating hemophilia. Circ Res 2001; 88: 137-144.
-
(2001)
Circ. Res.
, vol.88
, pp. 137-144
-
-
High, K.A.1
-
3
-
-
0034923893
-
Gene therapy in hemophilia: Clinical trials update
-
White II GC. Gene therapy in hemophilia: clinical trials update. Thromb Haemost 2001; 86: 172-177.
-
(2001)
Thromb. Haemost.
, vol.86
, pp. 172-177
-
-
White II, G.C.1
-
4
-
-
0029850534
-
Complete short-term correction of canine hemophilia A by in vivo gene therapy
-
Connelly S et al. Complete short-term correction of canine hemophilia A by in vivo gene therapy. Blood 1996; 88: 3846-3853.
-
(1996)
Blood
, vol.88
, pp. 3846-3853
-
-
Connelly, S.1
-
5
-
-
0032080179
-
Sustained phenotypic correction of murine hemophilia A by in vivo gene therapy
-
Connelly S et al. Sustained phenotypic correction of murine hemophilia A by in vivo gene therapy. Blood 1998; 91: 3273-3281.
-
(1998)
Blood
, vol.91
, pp. 3273-3281
-
-
Connelly, S.1
-
6
-
-
0033539486
-
Short-term correction of factor VIII deficiency in a murine model of hemophilia A after delivery of adenovirus murine factor VIII in utero
-
Lipshutz GS et al. Short-term correction of factor VIII deficiency in a murine model of hemophilia A after delivery of adenovirus murine factor VIII in utero. Proc Natl Acad Sci USA 1999; 96: 13324-13329.
-
(1999)
Proc. Natl. Acad. Sci. USA
, vol.96
, pp. 13324-13329
-
-
Lipshutz, G.S.1
-
7
-
-
12944317288
-
Sustained high-level expression of full-length human factor VIII and restoration of clotting activity in hemophilic mice using a minimal adenovirus vector
-
Balagué C et al. Sustained high-level expression of full-length human factor VIII and restoration of clotting activity in hemophilic mice using a minimal adenovirus vector. Blood 2000; 95: 820-828.
-
(2000)
Blood
, vol.95
, pp. 820-828
-
-
Balagué, C.1
-
8
-
-
0034630337
-
Partial correction of murine hemophilia A with neo-antigenic murine factor VIII
-
Sarkar R et al. Partial correction of murine hemophilia A with neo-antigenic murine factor VIII. Hum Gene Ther 2000; 11: 881-894.
-
(2000)
Hum. Gene Ther.
, vol.11
, pp. 881-894
-
-
Sarkar, R.1
-
9
-
-
0034814009
-
Adenovirus-mediated factor VIII gene expression results in attenuated anti-factor VIII-specific immunity in hemophilia A mice compared with factor VIII protein infusion
-
Bristol JA et al. Adenovirus-mediated factor VIII gene expression results in attenuated anti-factor VIII-specific immunity in hemophilia A mice compared with factor VIII protein infusion. Hum Gene Ther 2001; 12: 1651-1661.
-
(2001)
Hum. Gene Ther.
, vol.12
, pp. 1651-1661
-
-
Bristol, J.A.1
-
10
-
-
0035174331
-
Systemic delivery of an adenoviral vector encoding canine factor VIII results in short-term phenotypic correction, inhibitor development, and biphasic liver toxicity in hemophilia A dogs
-
Gallo-Penn AM et al. Systemic delivery of an adenoviral vector encoding canine factor VIII results in short-term phenotypic correction, inhibitor development, and biphasic liver toxicity in hemophilia A dogs. Blood 2001; 97: 107-113.
-
(2001)
Blood
, vol.97
, pp. 107-113
-
-
Gallo-Penn, A.M.1
-
11
-
-
0037372398
-
Therapeutic factor VIII levels and negligible toxicity in mouse and dog models of hemophilia A following gene therapy with high-capacity adenoviral vectors
-
Chuah MK et al. Therapeutic factor VIII levels and negligible toxicity in mouse and dog models of hemophilia A following gene therapy with high-capacity adenoviral vectors. Blood 2002; 101: 1734-1743.
-
(2002)
Blood
, vol.101
, pp. 1734-1743
-
-
Chuah, M.K.1
-
12
-
-
0033607305
-
Coexpression of factor VIII heavy and light chain adeno-associated viral vectors produces biologically active protein
-
Burton M et al. Coexpression of factor VIII heavy and light chain adeno-associated viral vectors produces biologically active protein. Proc Natl Acad Sci USA 1999; 96: 12725-12730.
-
(1999)
Proc. Natl. Acad. Sci. USA
, vol.96
, pp. 12725-12730
-
-
Burton, M.1
-
13
-
-
0034161486
-
Sustained expression of human factor VIII in mice using a parvovirus-based vector
-
Chao H, Mao L, Bruce AT, Walsh CE. Sustained expression of human factor VIII in mice using a parvovirus-based vector. Blood 2000; 95: 1594-1599.
-
(2000)
Blood
, vol.95
, pp. 1594-1599
-
-
Chao, H.1
Mao, L.2
Bruce, A.T.3
Walsh, C.E.4
-
14
-
-
0035874523
-
Induction of tolerance to human factor VIII in mice
-
Chao H, Walsh CE. Induction of tolerance to human factor VIII in mice. Blood 2001; 97: 3311-3312.
-
(2001)
Blood
, vol.97
, pp. 3311-3312
-
-
Chao, H.1
Walsh, C.E.2
-
15
-
-
0035988996
-
Expression of human factor VIII by splicing between dimerized AAV vectors
-
Chao H et al. Expression of human factor VIII by splicing between dimerized AAV vectors. Mol Ther 2002; 5: 716-722.
-
(2002)
Mol. Ther.
, vol.5
, pp. 716-722
-
-
Chao, H.1
-
16
-
-
0036377825
-
Evaluation of the duration of human factor VIII expression in nonhuman primates after systemic delivery of an adenoviral vector
-
Andrews JL et al. Evaluation of the duration of human factor VIII expression in nonhuman primates after systemic delivery of an adenoviral vector. Hum Gene Ther 2002; 13: 1331-1336.
-
(2002)
Hum. Gene Ther.
, vol.13
, pp. 1331-1336
-
-
Andrews, J.L.1
-
17
-
-
0033621126
-
Long-term expression of human coagulation factor VIII and correction of hemophilia A after in vivo retroviral gene transfer in factor VIII-deficient mice
-
VandenDriessche T et al. Long-term expression of human coagulation factor VIII and correction of hemophilia A after in vivo retroviral gene transfer in factor VIII-deficient mice. Proc Natl Acad Sci USA 1999; 96: 10379-10384.
-
(1999)
Proc. Natl. Acad. Sci. USA
, vol.96
, pp. 10379-10384
-
-
VandenDriessche, T.1
-
18
-
-
0034254320
-
Therapeutic levels of human factor VIII and IX using HIV-1-based lentiviral vectors in mouse liver
-
Park F, Ohashi K, Kay MA. Therapeutic levels of human factor VIII and IX using HIV-1-based lentiviral vectors in mouse liver. Blood 2000; 96: 1173-1176.
-
(2000)
Blood
, vol.96
, pp. 1173-1176
-
-
Park, F.1
Ohashi, K.2
Kay, M.A.3
-
19
-
-
0034960875
-
In vivo treatment of hemophilia A and mucopolysaccharidosis type VII using nonprimate lentiviral vectors
-
Stein CS et al. In vivo treatment of hemophilia A and mucopolysaccharidosis type VII using nonprimate lentiviral vectors. Mol Ther 2001; 3: 850-856.
-
(2001)
Mol. Ther.
, vol.3
, pp. 850-856
-
-
Stein, C.S.1
-
20
-
-
0036682975
-
Lentiviral vectors containing the human immunodeficiency virus type-1 central polypurine tract can efficiently transduce nondividing hepatocytes and antigen-presenting cells in vivo
-
VandenDriessche T et al. Lentiviral vectors containing the human immunodeficiency virus type-1 central polypurine tract can efficiently transduce nondividing hepatocytes and antigen-presenting cells in vivo. Blood 2002; 100: 813-822.
-
(2002)
Blood
, vol.100
, pp. 813-822
-
-
VandenDriessche, T.1
-
21
-
-
0027283419
-
Toward gene therapy for hemophilia A: Long-term persistence of factor VIII-secreting fibroblasts after transplantation into immunodeficient mice
-
Hoeben RC et al. Toward gene therapy for hemophilia A: long-term persistence of factor VIII-secreting fibroblasts after transplantation into immunodeficient mice. Hum Gene Ther 1993; 4: 179-186.
-
(1993)
Hum. Gene Ther.
, vol.4
, pp. 179-186
-
-
Hoeben, R.C.1
-
22
-
-
0028842288
-
Gene therapy for hemophilia A: Production of therapeutic levels of human factor VIII in vivo in mice
-
Dwarki VJ et al. Gene therapy for hemophilia A: production of therapeutic levels of human factor VIII in vivo in mice. Proc Natl Acad Sci USA 1995; 92: 1023-1027.
-
(1995)
Proc. Natl. Acad. Sci. USA
, vol.92
, pp. 1023-1027
-
-
Dwarki, V.J.1
-
23
-
-
0035822005
-
Nonviral transfer of the gene encoding coagulation factor VIII in patients with severe hemophilia A
-
Roth DA et al. Nonviral transfer of the gene encoding coagulation factor VIII in patients with severe hemophilia A. N Engl J Med 2001; 344: 1735-1742.
-
(2001)
N. Engl. J. Med.
, vol.344
, pp. 1735-1742
-
-
Roth, D.A.1
-
24
-
-
0032501988
-
Bone marrow stromal cells as targets for gene therapy of hemophilia A
-
Chuah MK et al. Bone marrow stromal cells as targets for gene therapy of hemophilia A. Hum Gene Ther 1998; 9: 353-365.
-
(1998)
Hum. Gene Ther.
, vol.9
, pp. 353-365
-
-
Chuah, M.K.1
-
25
-
-
0034689505
-
Long-term persistence of human bone marrow stromal cells transduced with factor VIII-retroviral vectors and transient production of therapeutic levels of human factor VIII in nomnyeloablated inummodeficient mice
-
Chuah MK et al. Long-term persistence of human bone marrow stromal cells transduced with factor VIII-retroviral vectors and transient production of therapeutic levels of human factor VIII in nomnyeloablated inummodeficient mice. Hum Gene Ther 2000; 11: 729-738.
-
(2000)
Hum. Gene Ther.
, vol.11
, pp. 729-738
-
-
Chuah, M.K.1
-
26
-
-
0026534020
-
Toward gene therapy in haemophilia A: Retrovirus-mediated transfer of a factor VIII gene into murine haematopoietic progenitor cells
-
Hoeben RC et al. Toward gene therapy in haemophilia A: retrovirus-mediated transfer of a factor VIII gene into murine haematopoietic progenitor cells. Thromb Haemost 1992; 67: 341-345.
-
(1992)
Thromb. Haemost.
, vol.67
, pp. 341-345
-
-
Hoeben, R.C.1
-
27
-
-
0032510756
-
Genetic induction of immune tolerance to human clotting factor VIII in a mouse model for hemophilia A
-
Evans GL, Morgan RA. Genetic induction of immune tolerance to human clotting factor VIII in a mouse model for hemophilia A. Proc Natl Acad Sci USA 1998; 95: 5734-5739.
-
(1998)
Proc. Natl. Acad. Sci. USA
, vol.95
, pp. 5734-5739
-
-
Evans, G.L.1
Morgan, R.A.2
-
28
-
-
0034839787
-
Tissue distribution of factor VIII gene expression in vivo - A closer look
-
Hollestelle MJ et al. Tissue distribution of factor VIII gene expression in vivo - a closer look. Thromb Haemost 2001; 86: 855-861.
-
(2001)
Thromb. Haemost.
, vol.86
, pp. 855-861
-
-
Hollestelle, M.J.1
-
29
-
-
0032578385
-
HIV, but not murine leukemia virus, vectors mediate high efficiency gene transfer into freshly isolated G0/G1 human hematopoietic stem cells
-
Uchida N et al. 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 1998; 95: 11939-11944.
-
(1998)
Proc. Natl. Acad. Sci. USA
, vol.95
, pp. 11939-11944
-
-
Uchida, N.1
-
30
-
-
0033613827
-
Transduction of human CD34+ cells that mediate long-term engraftment of NCOD/SCID mice by HIV vectors
-
Miyoshi H et al. Transduction of human CD34+ cells that mediate long-term engraftment of NCOD/SCID mice by HIV vectors. Science 1999; 283: 682-686.
-
(1999)
Science
, vol.283
, pp. 682-686
-
-
Miyoshi, H.1
-
31
-
-
0033542427
-
Human cord blood CD34+CD38- cell transduction via lentivirus-based gene transfer vectors
-
Evans JT, Kelly PF, O'Neill E, Garcia JV. Human cord blood CD34+CD38- cell transduction via lentivirus-based gene transfer vectors. Hum Gene Ther 1999; 10: 1479-1489.
-
(1999)
Hum. Gene Ther.
, vol.10
, pp. 1479-1489
-
-
Evans, J.T.1
Kelly, P.F.2
O'Neill, E.3
Garcia, J.V.4
-
32
-
-
13044309472
-
Stable transduction of quiescent CD34(+)CD38(-) human hematopoietic cells by HIV-1-based lentiviral vectors
-
Case SS et al. Stable transduction of quiescent CD34(+)CD38(-) human hematopoietic cells by HIV-1-based lentiviral vectors. Proc Natl Acad Sci USA 1999; 96: 2988-2993.
-
(1999)
Proc. Natl. Acad. Sci. USA
, vol.96
, pp. 2988-2993
-
-
Case, S.S.1
-
33
-
-
0037079742
-
+ NOD/SCID-repopulating cells
-
+ NOD/SCID-repopulating cells. Blood 2002; 99: 709-712.
-
(2002)
Blood
, vol.99
, pp. 709-712
-
-
Scherr, M.1
-
34
-
-
0034646513
-
HIV-1 genome nuclear import is mediated by a central DNA flap
-
Zennou V et al. HIV-1 genome nuclear import is mediated by a central DNA flap. Cell 2000; 101: 173-185.
-
(2000)
Cell
, vol.101
, pp. 173-185
-
-
Zennou, V.1
-
35
-
-
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 et al. 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 2000; 96: 4103-4110.
-
(2000)
Blood
, vol.96
, pp. 4103-4110
-
-
Sirven, A.1
-
36
-
-
0035013429
-
The HIV-1 DNA flap stimulates HIV vector-mediated cell transduction in the brain
-
Zennou V et al. The HIV-1 DNA flap stimulates HIV vector-mediated cell transduction in the brain. Nat Biotechnol 2001; 19: 446-450.
-
(2001)
Nat. Biotechnol.
, vol.19
, pp. 446-450
-
-
Zennou, V.1
-
37
-
-
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 MA. Modified HIV-1 based lentiviral vectors have an effect on viral transduction efficiency and gene expression in vitro and in vivo. Mol Ther 2001; 4: 164-173.
-
(2001)
Mol. Ther.
, vol.4
, pp. 164-173
-
-
Park, F.1
Kay, M.A.2
-
38
-
-
0034663381
-
Lentiviral-mediated gene transfer into human lymphocytes: Role of HIV-1 accessory proteins
-
Chinnasamy D et al. Lentiviral-mediated gene transfer into human lymphocytes: role of HIV-1 accessory proteins. Blood 2000; 96: 1309-1316.
-
(2000)
Blood
, vol.96
, pp. 1309-1316
-
-
Chinnasamy, D.1
-
39
-
-
0028201732
-
Tolerance, danger, and the extended family
-
Matzinger P. Tolerance, danger, and the extended family. Annu Rev Immunol 1994; 12: 991-1045.
-
(1994)
Annu. Rev. Immunol.
, vol.12
, pp. 991-1045
-
-
Matzinger, P.1
-
40
-
-
0037103167
-
Dangerous liaisons: The role of 'danger' signals in the immune response to gene therapy
-
Brown BD, Lillicrap D. Dangerous liaisons: the role of 'danger' signals in the immune response to gene therapy. Blood 2002; 100: 1133-1140.
-
(2002)
Blood
, vol.100
, pp. 1133-1140
-
-
Brown, B.D.1
Lillicrap, D.2
-
41
-
-
0034199170
-
Introduction of a xenogeneic gene via hematopoietic stem cells leads to specific tolerance in a rhesus monkey model
-
Heim DA et al. Introduction of a xenogeneic gene via hematopoietic stem cells leads to specific tolerance in a rhesus monkey model. Mol Ther 2000; 1: 533-544.
-
(2000)
Mol. Ther.
, vol.1
, pp. 533-544
-
-
Heim, D.A.1
-
42
-
-
0034809177
-
In vivo persistence of retrovirally transduced murine long-term repopulating cells is not limited by expression of foreign gene products in the fully or minimally myeloablated setting
-
Kang E et al. In vivo persistence of retrovirally transduced murine long-term repopulating cells is not limited by expression of foreign gene products in the fully or minimally myeloablated setting. Hum Gene Ther 2001; 12: 1663-1672.
-
(2001)
Hum. Gene Ther.
, vol.12
, pp. 1663-1672
-
-
Kang, E.1
-
43
-
-
0031884476
-
The canine factor VIII cDNA and 5′ flanking sequence
-
Cameron C et al. The canine factor VIII cDNA and 5′ flanking sequence. Thromb Haemost 1998; 79: 317-322.
-
(1998)
Thromb. Haemost.
, vol.79
, pp. 317-322
-
-
Cameron, C.1
-
44
-
-
0029996147
-
In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector
-
Naldini L et al. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 1996; 272: 263-267.
-
(1996)
Science
, vol.272
, pp. 263-267
-
-
Naldini, L.1
-
45
-
-
0029993858
-
Efficient transfer, integration, and sustained long-term expression of the transgene in adult rat brains injected with a lentiviral vector
-
Naldini L et al. Efficient transfer, integration, and sustained long-term expression of the transgene in adult rat brains injected with a lentiviral vector. Proc Natl Acad Sci USA 1996; 93: 11382-11388.
-
(1996)
Proc. Natl. Acad. Sci. USA
, vol.93
, pp. 11382-11388
-
-
Naldini, L.1
-
46
-
-
0030819379
-
Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo
-
Zufferey R et al. Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo. Nat Biotechnol 1997; 15: 871-875.
-
(1997)
Nat. Biotechnol.
, vol.15
, pp. 871-875
-
-
Zufferey, R.1
-
47
-
-
0034857455
-
Quantitative determination of lentiviral vector particle numbers by real-time PCR
-
Scherr M et al. Quantitative determination of lentiviral vector particle numbers by real-time PCR. Biotechniques 2001; 31: 520-526.
-
(2001)
Biotechniques
, vol.31
, pp. 520-526
-
-
Scherr, M.1
-
48
-
-
0032845686
-
Monitoring of BCR-ABL expression using real-time RT-PCR in CML after bone marrow or peripheral blood stem cell transplantation
-
Eder M et al. Monitoring of BCR-ABL expression using real-time RT-PCR in CML after bone marrow or peripheral blood stem cell transplantation. Leukemia 1999; 13: 1383-1389.
-
(1999)
Leukemia
, vol.13
, pp. 1383-1389
-
-
Eder, M.1
-
49
-
-
0342470476
-
Quantitative assessment of retroviral transfer of the human multidrug resistance 1 gene to human mobilized peripheral blood progenitor cells engrafted in nonobese diabetic/severe combined immunodeficient mice
-
Schiedlmeier B et al. Quantitative assessment of retroviral transfer of the human multidrug resistance 1 gene to human mobilized peripheral blood progenitor cells engrafted in nonobese diabetic/severe combined immunodeficient mice. Blood 2000; 95: 1237-1248.
-
(2000)
Blood
, vol.95
, pp. 1237-1248
-
-
Schiedlmeier, B.1
|