-
1
-
-
0035135747
-
Viral vectors for gene therapy: The art of turning infectious agents into vehicles of therapeutics
-
Kay MA, Glorioso JC, Naldini L. Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics. Nat Med. 2001; 7:33-40.
-
(2001)
Nat Med
, vol.7
, pp. 33-40
-
-
Kay, M.A.1
Glorioso, J.C.2
Naldini, L.3
-
2
-
-
0035676918
-
Gene therapy for haemophilia
-
Pasi KJ. Gene therapy for haemophilia. Br J Haematol. 2001;115:744-757.
-
(2001)
Br J Haematol
, vol.115
, pp. 744-757
-
-
Pasi, K.J.1
-
3
-
-
0037325357
-
Gene transfer as an approach to treating hemophilia
-
High KA. Gene transfer as an approach to treating hemophilia. Semin Thromb Hemost. 2003;29: 107-120.
-
(2003)
Semin Thromb Hemost
, vol.29
, pp. 107-120
-
-
High, K.A.1
-
4
-
-
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
-
5
-
-
0036776745
-
Antigen degradation or presentation by MHC class I molecules via classical and non-classical pathways
-
Gromme M, Neefjes J. Antigen degradation or presentation by MHC class I molecules via classical and non-classical pathways. Mol Immunol. 2002;39:181-202.
-
(2002)
Mol Immunol
, vol.39
, pp. 181-202
-
-
Gromme, M.1
Neefjes, J.2
-
6
-
-
0038446697
-
Humoral immune response in mice against a circulating antigen induced by adenoviral transfer is strictly dependent on expression in antigen-presenting cells
-
De Geest BR, Van Linthout SA, Collen D. Humoral immune response in mice against a circulating antigen induced by adenoviral transfer is strictly dependent on expression in antigen-presenting cells. Blood. 2003;101:2551-2556.
-
(2003)
Blood
, vol.101
, pp. 2551-2556
-
-
De Geest, B.R.1
Van Linthout, S.A.2
Collen, D.3
-
7
-
-
0035080378
-
Intravenous administration of an E1/E3-deleted adenoviral vector induces tolerance to factor IX in C57BL/6 mice
-
Fields PA, Armstrong E, Hagstrom JN, et al. Intravenous administration of an E1/E3-deleted adenoviral vector induces tolerance to factor IX in C57BL/6 mice. Gene Ther. 2001;8:354-361.
-
(2001)
Gene Ther
, vol.8
, pp. 354-361
-
-
Fields, P.A.1
Armstrong, E.2
Hagstrom, J.N.3
-
8
-
-
0034151775
-
Role of vector in activation of T cell subsets in immune responses against the secreted transgene product factor IX
-
Fields PA, Kowalczyk DW, Arruda VR, et al. Role of vector in activation of T cell subsets in immune responses against the secreted transgene product factor IX. Mol Ther. 2000;1:225-235.
-
(2000)
Mol Ther
, vol.1
, pp. 225-235
-
-
Fields, P.A.1
Kowalczyk, D.W.2
Arruda, V.R.3
-
9
-
-
0036376642
-
Influence of vector dose on factor IX-specific T and B cell responses in muscle-directed gene therapy
-
Herzog RW, Fields PA, Arruda VR, et al. Influence of vector dose on factor IX-specific T and B cell responses in muscle-directed gene therapy. Hum Gene Ther. 2002;13:1281-1291.
-
(2002)
Hum Gene Ther
, vol.13
, pp. 1281-1291
-
-
Herzog, R.W.1
Fields, P.A.2
Arruda, V.R.3
-
10
-
-
0030999469
-
Adenovirus-mediated gene transfer: Influence of transgene, mouse strain and type of immune response on persistence of transgene expression
-
Michou AI, Santoro L, Christ M, Julliard V, Pavirani A, Mehtali M. Adenovirus-mediated gene transfer: influence of transgene, mouse strain and type of immune response on persistence of transgene expression. Gene Ther. 1997;4:473-482.
-
(1997)
Gene Ther
, vol.4
, pp. 473-482
-
-
Michou, A.I.1
Santoro, L.2
Christ, M.3
Julliard, V.4
Pavirani, A.5
Mehtali, M.6
-
11
-
-
0036171327
-
Efficient gene delivery and targeted expression to hepatocytes in vivo by improved lentiviral vectors
-
Follenzi A, Sabatino G, Lombardo A, Boccaccio C, Naldini L. 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
Boccaccio, C.4
Naldini, L.5
-
12
-
-
0034989411
-
Transduction of liver cells by lentiviral vectors: Analysis in living animals by fluorescence imaging
-
Pfeifer A, Kessler T, Yang M, et al. Transduction of liver cells by lentiviral vectors: analysis in living animals by fluorescence imaging. Mol Ther. 2001; 3:319-322.
-
(2001)
Mol Ther
, vol.3
, pp. 319-322
-
-
Pfeifer, A.1
Kessler, T.2
Yang, M.3
-
13
-
-
0036682975
-
Lentiviral vectors containing the human immuno-deficiency virus type-1 central polypurine tract can efficiently transduce nondividing hepatocytes and antigen-presenting cells in vivo
-
Vandendriessche T, Thorrez L, Naldiril L, et al. Lentiviral vectors containing the human immuno-deficiency 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
Thorrez, L.2
Naldiril, L.3
-
14
-
-
0036137639
-
Production of human clotting Factor IX without toxicity in mice after vascular delivery of a lentiviral vector
-
Tsui LV, Kelly M, Zayek N, et al. Production of human clotting Factor IX without toxicity in mice after vascular delivery of a lentiviral vector. Nat Biotechnol. 2002;20:53-57.
-
(2002)
Nat Biotechnol
, vol.20
, pp. 53-57
-
-
Tsui, L.V.1
Kelly, M.2
Zayek, N.3
-
15
-
-
0034254320
-
Therapeutic levels of human factor VIII and IX using HIV-1-based lenti-viral vectors in mouse liver
-
Park F, Ohashi K, Kay MA. Therapeutic levels of human factor VIII and IX using HIV-1-based lenti-viral 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
-
16
-
-
0037853196
-
Efficient production of human FVIII in hemophilic mice using lentiviral vectors
-
Kootstra NA, Matsumura R, Verma IM. Efficient production of human FVIII in hemophilic mice using lentiviral vectors. Mol Ther. 2003;7:623-631.
-
(2003)
Mol Ther
, vol.7
, pp. 623-631
-
-
Kootstra, N.A.1
Matsumura, R.2
Verma, I.M.3
-
17
-
-
0033694598
-
Universal GFP reporter for the study of vascular development
-
Motoike T, Loughna S, Perens E, et al. Universal GFP reporter for the study of vascular development. Genesis. 2000;28:75-81.
-
(2000)
Genesis
, vol.28
, pp. 75-81
-
-
Motoike, T.1
Loughna, S.2
Perens, E.3
-
18
-
-
0038644403
-
Targeting exogenous genes to tumor angiogenesis by transplantation of genetically modified hematopoietic stem cells
-
De Palma M, Venneri MA, Roca C, Naldini L. Targeting exogenous genes to tumor angiogenesis by transplantation of genetically modified hematopoietic stem cells. Nat Med. 2003;9:789-795.
-
(2003)
Nat Med
, vol.9
, pp. 789-795
-
-
De Palma, M.1
Venneri, M.A.2
Roca, C.3
Naldini, L.4
-
19
-
-
0035860585
-
Cytotoxic T cell polyepitope vaccines delivered by ISCOMs
-
Le TT, Drane D, Malliaros J, et al. Cytotoxic T cell polyepitope vaccines delivered by ISCOMs. Vaccine. 2001;19:4669-4675.
-
(2001)
Vaccine
, vol.19
, pp. 4669-4675
-
-
Le, T.T.1
Drane, D.2
Malliaros, J.3
-
20
-
-
0033203120
-
Selective transport of internalized antigens to the cytosol for MHC class I presentation in dendritic cells
-
Rodriguez A, Regnault A, Klaijmeer M, Ricciardi-Castagnoli P, Amigorena S. Selective transport of internalized antigens to the cytosol for MHC class I presentation in dendritic cells. Nat Cell Biol. 1999;1:362-366.
-
(1999)
Nat Cell Biol
, vol.1
, pp. 362-366
-
-
Rodriguez, A.1
Regnault, A.2
Klaijmeer, M.3
Ricciardi-Castagnoli, P.4
Amigorena, S.5
-
21
-
-
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: Nonobese diabetic/severe combined immunodeficient
-
Placibello 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: nonobese diabetic/severe combined immunodeficient. Blood. 2002;100:4391-4400.
-
(2002)
Blood
, vol.100
, pp. 4391-4400
-
-
Placibello, W.1
Bruno, S.2
Sanavio, F.3
-
22
-
-
0029873751
-
On immunological memory
-
Zinkernagel RM, Bachmann MF, Kundig TM, Oehen S, Pirchet H, Hengartner H. On immunological memory. Annu Rev Immunol. 1996;14:333-367.
-
(1996)
Annu Rev Immunol
, vol.14
, pp. 333-367
-
-
Zinkernagel, R.M.1
Bachmann, M.F.2
Kundig, T.M.3
Oehen, S.4
Pirchet, H.5
Hengartner, H.6
-
23
-
-
0034853102
-
Risk and prevention of anti-factor IX formation in AAV-mediated gene transfer in the context of a large deletion of F9
-
Fields PA, Arruda VR, Armstrong E, et al. Risk and prevention of anti-factor IX formation in AAV-mediated gene transfer in the context of a large deletion of F9. Mol Ther. 2001;4:201-210.
-
(2001)
Mol Ther
, vol.4
, pp. 201-210
-
-
Fields, P.A.1
Arruda, V.R.2
Armstrong, E.3
-
24
-
-
0035704168
-
The enhanced green fluorescent protein (eGFP) is minimally immunogenic in C57BL/6 mice
-
Skelton D, Satake N, Kohn DB. The enhanced green fluorescent protein (eGFP) is minimally immunogenic in C57BL/6 mice. Gene Ther. 2001;8: 1813-1814.
-
(2001)
Gene Ther
, vol.8
, pp. 1813-1814
-
-
Skelton, D.1
Satake, N.2
Kohn, D.B.3
-
25
-
-
0035700549
-
EGFP-transduced EL-4 cells from tumors in C57BL/6 mice
-
Denaro M, Oldmixon B, Patience C, Andersson G, Down J. EGFP-transduced EL-4 cells from tumors in C57BL/6 mice. Gene Ther. 2001;8:1814-1815.
-
(2001)
Gene Ther
, vol.8
, pp. 1814-1815
-
-
Denaro, M.1
Oldmixon, B.2
Patience, C.3
Andersson, G.4
Down, J.5
-
26
-
-
0028914687
-
Strain related variations in adenovirally mediated transgene expression from mouse hepatocytes in vivo: Comparisons between immunocompetent and immunodeficient inbred strains
-
Barr D, Tubb J, Ferguson D, et al. Strain related variations in adenovirally mediated transgene expression from mouse hepatocytes in vivo: comparisons between immunocompetent and immunodeficient inbred strains. Gene Ther. 1995;2: 151-155.
-
(1995)
Gene Ther
, vol.2
, pp. 151-155
-
-
Barr, D.1
Tubb, J.2
Ferguson, D.3
-
27
-
-
0031969428
-
Transduction of dendritic cells by DNA viral vectors directs the immune response to transgene products in muscle fibers
-
Jooss K, Yang Y, Fisher KJ, Wilson JM. Transduction of dendritic cells by DNA viral vectors directs the immune response to transgene products in muscle fibers. Proc Natl Acad Sci U S A. 1998; 72:4212-4223.
-
(1998)
Proc Natl Acad Sci U S A
, vol.72
, pp. 4212-4223
-
-
Jooss, K.1
Yang, Y.2
Fisher, K.J.3
Wilson, J.M.4
-
28
-
-
0036288752
-
Efficient transduction of dendritic cells and induction of a T-cell response by third-generation lentivectors
-
Esslinger C, Romero P, MacDonald HR. Efficient transduction of dendritic cells and induction of a T-cell response by third-generation lentivectors. Hum Gene Ther. 2002;13:1091-1100.
-
(2002)
Hum Gene Ther
, vol.13
, pp. 1091-1100
-
-
Esslinger, C.1
Romero, P.2
MacDonald, H.R.3
-
29
-
-
0032812877
-
Immune response to green fluorescent protein: Implications for gene therapy
-
Stripecke R, Carmen Villacres M, et al. Immune response to green fluorescent protein: implications for gene therapy. Gene Ther. 1999;6:1305-1312.
-
(1999)
Gene Ther
, vol.6
, pp. 1305-1312
-
-
Stripecke, R.1
Carmen Villacres, M.2
-
30
-
-
0037066427
-
The danger model: A renewed sense of self
-
Matzinger P. The danger model: a renewed sense of self. Science. 2002;296:301-305.
-
(2002)
Science
, vol.296
, pp. 301-305
-
-
Matzinger, P.1
-
31
-
-
0038819053
-
In vivo administration of a lentiviral vaccine targets DCs and induces efficient CD8(+) T cell responses
-
Esslinger C, Chapatte L, Finke D, et al. In vivo administration of a lentiviral vaccine targets DCs and induces efficient CD8(+) T cell responses. J Clin Invest. 2003;111:1673-1681.
-
(2003)
J Clin Invest
, vol.111
, pp. 1673-1681
-
-
Esslinger, C.1
Chapatte, L.2
Finke, D.3
-
32
-
-
0032605547
-
Expanded-capacity adenoviral vectors-the helper-dependent vectors
-
Morsy MA, Caskey CT. Expanded-capacity adenoviral vectors-the helper-dependent vectors. Mol Med Today. 1999;5:18-24.
-
(1999)
Mol Med Today
, vol.5
, pp. 18-24
-
-
Morsy, M.A.1
Caskey, C.T.2
-
33
-
-
0033587482
-
Use of a liver-specific promoter reduces immune response to the transgene in adenoviral vectors
-
Pastore L, Morral N, Zhou H, et al. Use of a liver-specific promoter reduces immune response to the transgene in adenoviral vectors. Hum Gene Ther. 1999;10:1773-1781.
-
(1999)
Hum Gene Ther
, vol.10
, pp. 1773-1781
-
-
Pastore, L.1
Morral, N.2
Zhou, H.3
-
34
-
-
0037988905
-
Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer
-
Mingozzi F, Liu YL, Dobrzynski E, et al. Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer. J Clin Invest. 2003;111:1347-1356.
-
(2003)
J Clin Invest
, vol.111
, pp. 1347-1356
-
-
Mingozzi, F.1
Liu, Y.L.2
Dobrzynski, E.3
-
35
-
-
0036592086
-
A new adenoviral helper-dependent vector results in long-term therapeutic levels of human coagulation factor IX at low doses in vivo
-
Ehrhardt A, Kay MA. A new adenoviral helper-dependent vector results in long-term therapeutic levels of human coagulation factor IX at low doses in vivo. Blood. 2002;99:3923-3930.
-
(2002)
Blood
, vol.99
, pp. 3923-3930
-
-
Ehrhardt, A.1
Kay, M.A.2
-
36
-
-
0141593513
-
A gene-deleted adenoviral vector results in phenotypic correction of canine hemophilia B without liver toxicity or thrombocytopenia
-
Ehrhardt A, Xu H, Dillow AM, Bellinger DA, Nichols TC, Kay MA. A gene-deleted adenoviral vector results in phenotypic correction of canine hemophilia B without liver toxicity or thrombocytopenia. Blood. 2003;102:2403-2411.
-
(2003)
Blood
, vol.102
, pp. 2403-2411
-
-
Ehrhardt, A.1
Xu, H.2
Dillow, A.M.3
Bellinger, D.A.4
Nichols, T.C.5
Kay, M.A.6
-
37
-
-
0037089331
-
Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy
-
Mount JD, Herzog RW, Tillson DM, et al. Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy. Blood. 2002;99:2670-2676.
-
(2002)
Blood
, vol.99
, pp. 2670-2676
-
-
Mount, J.D.1
Herzog, R.W.2
Tillson, D.M.3
-
39
-
-
0030997346
-
Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors
-
Snyder RO, Miao CH, Patijn GA, et al. Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors. Nat Genet. 1997;16:270-276.
-
(1997)
Nat Genet
, vol.16
, pp. 270-276
-
-
Snyder, R.O.1
Miao, C.H.2
Patijn, G.A.3
-
40
-
-
17444387438
-
Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector
-
Herzog RW, Yang EY, Couto LB, et al. Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector. Nat Med. 1999; 5:56-63.
-
(1999)
Nat Med
, vol.5
, pp. 56-63
-
-
Herzog, R.W.1
Yang, E.Y.2
Couto, L.B.3
-
41
-
-
17444372024
-
Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors
-
Snyder RO, Miao C, Meuse L, et al. Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors. Nat Med. 1999;5:64-70.
-
(1999)
Nat Med
, vol.5
, pp. 64-70
-
-
Snyder, R.O.1
Miao, C.2
Meuse, L.3
-
42
-
-
0032762405
-
Adenovirus-mediated expression of human coagulation factor IX in the rhesus macaque is associated with dose-limiting toxicity
-
Lozier JN, Metzger ME, Donahue RE, Morgan RA. Adenovirus-mediated expression of human coagulation factor IX in the rhesus macaque is associated with dose-limiting toxicity. Blood. 1999;94:3968-3975.
-
(1999)
Blood
, vol.94
, pp. 3968-3975
-
-
Lozier, J.N.1
Metzger, M.E.2
Donahue, R.E.3
Morgan, R.A.4
-
43
-
-
0034137797
-
Sustained expression of therapeutic level of factor IX in hemophilia B dogs by AAV-mediated gene therapy in liver
-
Wang L, Nichols TC, Read MS, Bellinger DA, Verma IM. Sustained expression of therapeutic level of factor IX in hemophilia B dogs by AAV-mediated gene therapy in liver. Mol Ther. 2000;1: 154-158.
-
(2000)
Mol Ther
, vol.1
, pp. 154-158
-
-
Wang, L.1
Nichols, T.C.2
Read, M.S.3
Bellinger, D.A.4
Verma, I.M.5
-
44
-
-
0034050904
-
Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector
-
Kay MA, Manno CS, Ragni MV, et al. Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. Nat Genet. 2000;24:257-261.
-
(2000)
Nat Genet
, vol.24
, pp. 257-261
-
-
Kay, M.A.1
Manno, C.S.2
Ragni, M.V.3
-
45
-
-
0013442744
-
AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B
-
Manno CS, Chew AJ, Hutchison S, et al. AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood. 2003;101:2963-2972.
-
(2003)
Blood
, vol.101
, pp. 2963-2972
-
-
Manno, C.S.1
Chew, A.J.2
Hutchison, S.3
-
46
-
-
0034849318
-
Muscle-directed gene transfer and transient immune suppression result in sustained partial correction of canine hemophilia B caused by a null mutation
-
Herzog RW, Mount JD, Arruda VR, High KA, Lothrop CD Jr. Muscle-directed gene transfer and transient immune suppression result in sustained partial correction of canine hemophilia B caused by a null mutation. Mol Ther. 2001;4:192-200.
-
(2001)
Mol Ther
, vol.4
, pp. 192-200
-
-
Herzog, R.W.1
Mount, J.D.2
Arruda, V.R.3
High, K.A.4
Lothrop Jr., C.D.5
-
47
-
-
13044304200
-
Administration of helper-dependent adenoviral vectors and sequential delivery of different vector serotype for long-term liver-directed gene transfer in baboons
-
Morral N, O'Neal W, Rice K, et al. Administration of helper-dependent adenoviral vectors and sequential delivery of different vector serotype for long-term liver-directed gene transfer in baboons. Proc Natl Acad Sci U S A. 1999;96:12816-12821.
-
(1999)
Proc Natl Acad Sci U S A
, vol.96
, pp. 12816-12821
-
-
Morral, N.1
O'Neal, W.2
Rice, K.3
-
48
-
-
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, Schiedner G, Thorrez L, 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. 2003;101:1734-1743.
-
(2003)
Blood
, vol.101
, pp. 1734-1743
-
-
Chuah, M.K.1
Schiedner, G.2
Thorrez, L.3
|