-
1
-
-
0033091484
-
In utero gene therapy: The case for
-
Schneider H, Coutelle C. In utero gene therapy: The case for. Nature Med 1999; 5: 256-257.
-
(1999)
Nature Med.
, vol.5
, pp. 256-257
-
-
Schneider, H.1
Coutelle, C.2
-
3
-
-
0033121139
-
Prenatal gene therapy: Can the technical hurdles be overcome?
-
Senut M-C, Gage FH. Prenatal gene therapy: can the technical hurdles be overcome? Mol Med Today 1999; 5: 152-156.
-
(1999)
Mol. Med. Today
, vol.5
, pp. 152-156
-
-
Senut, M.-C.1
Gage, F.H.2
-
4
-
-
0032821708
-
Prospects for in utero human gene therapy
-
Zanjani ED, Anderson WF. Prospects for in utero human gene therapy. Science 1999; 285: 2084-2088.
-
(1999)
Science
, vol.285
, pp. 2084-2088
-
-
Zanjani, E.D.1
Anderson, W.F.2
-
5
-
-
14044270998
-
Gene therapy in utero: Experience and limitations
-
Beckmann MW, Dall P, Krüssel JS, Niederacher D, Tutschek B (eds). Steinkopff-Verlag: Darmstadt, in press
-
Schneider H, Coutelle C. Gene therapy in utero: experience and limitations. In Molecular Medicine in Obstetrics and Gynaecology, Beckmann MW, Dall P, Krüssel JS, Niederacher D, Tutschek B (eds). Steinkopff-Verlag: Darmstadt, in press.
-
Molecular Medicine in Obstetrics and Gynaecology
-
-
Schneider, H.1
Coutelle, C.2
-
6
-
-
0034180256
-
Recombinant DNA Advisory Committee Prenatal gene transfer: Scientific, medical and ethical issues. A report of the Recombinant DNA Advisory Committee
-
US National Institutes of Health
-
US National Institutes of Health. Recombinant DNA Advisory Committee Prenatal gene transfer: scientific, medical and ethical issues. A report of the Recombinant DNA Advisory Committee. Hum Gene Ther 2000; 11: 1211-1229.
-
(2000)
Hum. Gene Ther.
, vol.11
, pp. 1211-1229
-
-
-
7
-
-
0032768176
-
Successful expression of β-galactosidase and factor IX transgenes in fetal and neonatal sheep after ultrasound-guided percutaneous adenovirus vector administration into the umbilical vein
-
Themis M, Schneider H, Kiserud T, et al. Successful expression of β-galactosidase and factor IX transgenes in fetal and neonatal sheep after ultrasound-guided percutaneous adenovirus vector administration into the umbilical vein. Gene Ther 1999; 6: 1239-1248.
-
(1999)
Gene Ther.
, vol.6
, pp. 1239-1248
-
-
Themis, M.1
Schneider, H.2
Kiserud, T.3
-
8
-
-
0033222971
-
Therapeutic concentrations of human factor IX in mice after gene delivery into the amniotic cavity: A model for the prenatal treatment of haemophilia B
-
Schneider H, Adebakin S, Themis M, et al. Therapeutic concentrations of human factor IX in mice after gene delivery into the amniotic cavity: a model for the prenatal treatment of haemophilia B. J Gene Med 1999; 1: 424-432.
-
(1999)
J. Gene Med.
, vol.1
, pp. 424-432
-
-
Schneider, H.1
Adebakin, S.2
Themis, M.3
-
9
-
-
0029046114
-
Adenovirus-mediated gene transfer to fetal pulmonary epithelia in vitro and in vivo
-
McCray PB, Armstrong K, Zabner J, et al. Adenovirus-mediated gene transfer to fetal pulmonary epithelia in vitro and in vivo. J Clin Invest 1995; 95: 2620-2632.
-
(1995)
J. Clin. Invest.
, vol.95
, pp. 2620-2632
-
-
McCray, P.B.1
Armstrong, K.2
Zabner, J.3
-
10
-
-
0029127914
-
Adenovirus-mediated gene transfer to the respiratory tract of fetal sheep in utero
-
Vincent MC, Trapnell BC, Baughman RP, Wert SE, Whitsett JA, Iwamoto HS. Adenovirus-mediated gene transfer to the respiratory tract of fetal sheep in utero. Hum Gene Ther 1995; 6: 1019-1028.
-
(1995)
Hum. Gene Ther.
, vol.6
, pp. 1019-1028
-
-
Vincent, M.C.1
Trapnell, B.C.2
Baughman, R.P.3
Wert, S.E.4
Whitsett, J.A.5
Iwamoto, H.S.6
-
11
-
-
0028834921
-
In utero gene transfer into pulmonary epithelium
-
Sekhon HS, Larson EJ. In utero gene transfer into pulmonary epithelium. Nature Med 1995; 1: 1201-1203.
-
(1995)
Nature Med.
, vol.1
, pp. 1201-1203
-
-
Sekhon, H.S.1
Larson, E.J.2
-
12
-
-
0030867444
-
Foetal gene delivery in mice by intra-amniotic administration of retroviral producer cells and adenovirus
-
Douar AM, Adebakin S, Themis M, Pavirani A, Cook T, Coutelle C. Foetal gene delivery in mice by intra-amniotic administration of retroviral producer cells and adenovirus. Gene Ther 1997; 4: 883-890.
-
(1997)
Gene Ther.
, vol.4
, pp. 883-890
-
-
Douar, A.M.1
Adebakin, S.2
Themis, M.3
Pavirani, A.4
Cook, T.5
Coutelle, C.6
-
13
-
-
0030776948
-
Adenovirus-mediated gene transfer during initial organogenesis in the mammalian embryo is promoter-dependent and tissue-specific
-
Baldwin HS, Mickanin C, Buck C. Adenovirus-mediated gene transfer during initial organogenesis in the mammalian embryo is promoter-dependent and tissue-specific. Gene Ther 1997; 4: 1142-1149.
-
(1997)
Gene Ther.
, vol.4
, pp. 1142-1149
-
-
Baldwin, H.S.1
Mickanin, C.2
Buck, C.3
-
14
-
-
0030780567
-
In utero cardiac gene transfer via intraplacental delivery of recombinant adenovirus
-
Woo YJ, Raju GP, Swain JL, Richmond ME, Gardner TJ, Balice-Gordon RJ. In utero cardiac gene transfer via intraplacental delivery of recombinant adenovirus. Circulation 1997; 96: 3561-3569.
-
(1997)
Circulation
, vol.96
, pp. 3561-3569
-
-
Woo, Y.J.1
Raju, G.P.2
Swain, J.L.3
Richmond, M.E.4
Gardner, T.J.5
Balice-Gordon, R.J.6
-
15
-
-
0032551689
-
r gene in sheep after direct injection of retroviral vectors into preimmune fetuses
-
r gene in sheep after direct injection of retroviral vectors into preimmune fetuses. Hum Gene Ther 1998; 9: 1571-1585.
-
(1998)
Hum. Gene Ther.
, vol.9
, pp. 1571-1585
-
-
Porada, C.D.1
Tran, N.2
Eglitis, M.3
-
16
-
-
0032784954
-
Temporally regulated expression patterns following in utero adenovirus-mediated gene transfer
-
Schachtner SK, Buck CA, Bergelson JM, Baldwin HS. Temporally regulated expression patterns following in utero adenovirus-mediated gene transfer. Gene Ther 1999; 6: 1249-1257.
-
(1999)
Gene Ther.
, vol.6
, pp. 1249-1257
-
-
Schachtner, S.K.1
Buck, C.A.2
Bergelson, J.M.3
Baldwin, H.S.4
-
17
-
-
0033072104
-
Fetal gene therapy: Efficacy, toxicity, and immunologic effects of early gestation recombinant adenovirus
-
Yang EY, Cass DL, Sylvester KG, Wilson JM, Adzick NS. Fetal gene therapy: efficacy, toxicity, and immunologic effects of early gestation recombinant adenovirus. J Pediatr Surg 1999; 34: 235-241.
-
(1999)
J. Pediatr. Surg.
, vol.34
, pp. 235-241
-
-
Yang, E.Y.1
Cass, D.L.2
Sylvester, K.G.3
Wilson, J.M.4
Adzick, N.S.5
-
18
-
-
17644448979
-
Intrauterine gene transfer: Gestational stage-specific gene delivery in mice
-
Türkay A, Saunders TL, Kurachi K. Intrauterine gene transfer: gestational stage-specific gene delivery in mice. Gene Ther 1999; 6: 1685-1694.
-
(1999)
Gene Ther.
, vol.6
, pp. 1685-1694
-
-
Türkay, A.1
Saunders, T.L.2
Kurachi, K.3
-
20
-
-
0033373301
-
Prevalence and outcome of intracranial haemorrhage in haemophiliacs - A survey of the paediatric group of the German Society of Thrombosis and Haemostasis (GTH)
-
Klinge J, Auberger K, Auerswald G, Brackmann HH, Mauz-Korholz C, Kreuz W. Prevalence and outcome of intracranial haemorrhage in haemophiliacs - a survey of the paediatric group of the German Society of Thrombosis and Haemostasis (GTH). Eur J Pediatr 1999; 158 (Suppl 3): 162-165.
-
(1999)
Eur. J. Pediatr.
, vol.158
, Issue.SUPPL. 3
, pp. 162-165
-
-
Klinge, J.1
Auberger, K.2
Auerswald, G.3
Brackmann, H.H.4
Mauz-Korholz, C.5
Kreuz, W.6
-
21
-
-
0031925855
-
How do some haemophiliacs develop inhibitors?
-
Vermylen J. How do some haemophiliacs develop inhibitors? Haemophilia 1998; 4: 538-542.
-
(1998)
Haemophilia
, vol.4
, pp. 538-542
-
-
Vermylen, J.1
-
22
-
-
0027521773
-
Adenovirus-mediated expression of therapeutic plasma levels of human factor IX in mice
-
Smith TA, Mehaffey MG, Kayda DB, et al. Adenovirus-mediated expression of therapeutic plasma levels of human factor IX in mice. Nature Genet 1993; 5: 397-402.
-
(1993)
Nature Genet.
, vol.5
, pp. 397-402
-
-
Smith, T.A.1
Mehaffey, M.G.2
Kayda, D.B.3
-
23
-
-
0027686238
-
In vivo gene therapy of hemophilia B: Sustained partial correction in factor IX-deficient dogs
-
Kay MA, Rothenberg S, Landen CN, et al. In vivo gene therapy of hemophilia B: sustained partial correction in factor IX-deficient dogs. Science 1993; 262: 117-119.
-
(1993)
Science
, vol.262
, pp. 117-119
-
-
Kay, M.A.1
Rothenberg, S.2
Landen, C.N.3
-
24
-
-
0027325520
-
Regulation of the phosphoenolpyruvate carboxykinase/human factor IX gene introduced into the livers of adult rats by receptor-mediated gene transfer
-
Ferkol T, Lindberg GL, Chen J, et al. Regulation of the phosphoenolpyruvate carboxykinase/human factor IX gene introduced into the livers of adult rats by receptor-mediated gene transfer. FASEB J 1993; 7: 1081-1091.
-
(1993)
FASEB J.
, vol.7
, pp. 1081-1091
-
-
Ferkol, T.1
Lindberg, G.L.2
Chen, J.3
-
25
-
-
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. Nature Genet 1997; 16: 270-276.
-
(1997)
Nature Genet.
, vol.16
, pp. 270-276
-
-
Snyder, R.O.1
Miao, C.H.2
Patijn, G.A.3
-
26
-
-
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. Nature Med 1999; 5: 64-70.
-
(1999)
Nature Med.
, vol.5
, pp. 64-70
-
-
Snyder, R.O.1
Miao, C.2
Meuse, L.3
-
27
-
-
0034137797
-
Sustained expression of therapeutic levels of factor IX in hemophilia B dogs by AAV-mediated gene therapy in liver
-
Wang L, Nichols TC, Read MS, et al. Sustained expression of therapeutic levels 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
-
28
-
-
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. Nature Med 1999; 5: 56-63.
-
(1999)
Nature Med.
, vol.5
, pp. 56-63
-
-
Herzog, R.W.1
Yang, E.Y.2
Couto, L.B.3
-
30
-
-
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. Nature Genet 2000; 24: 257-261.
-
(2000)
Nature Genet.
, vol.24
, pp. 257-261
-
-
Kay, M.A.1
Manno, C.S.2
Ragni, M.V.3
-
31
-
-
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, Sarkar R, Flebbe-Rehwaldt L, Kazazian H, Gaensler KML. 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
Sarkar, R.2
Flebbe-Rehwaldt, L.3
Kazazian, H.4
Gaensler, K.M.L.5
-
32
-
-
2642642141
-
Production of high-titer recombinant adeno-associated virus vectors in the absence of helper virus
-
Xiao X, Li J, Samulski RJ. Production of high-titer recombinant adeno-associated virus vectors in the absence of helper virus. J Virol 1998; 72: 2224-2232.
-
(1998)
J. Virol.
, vol.72
, pp. 2224-2232
-
-
Xiao, X.1
Li, J.2
Samulski, R.J.3
-
33
-
-
0002988871
-
Production of recombinant adeno-associated viral vectors
-
Dracopoli N, Haines J, Krof B et al. (eds), John Wiley: New York
-
Snyder RO, Xiao X, Samulski RJ. Production of recombinant adeno-associated viral vectors. In Current Protocols in Human Genetics Dracopoli N, Haines J, Krof B et al. (eds), vol. 1. John Wiley: New York, 1996; 1-24.
-
(1996)
Current Protocols in Human Genetics
, vol.1
, pp. 1-24
-
-
Snyder, R.O.1
Xiao, X.2
Samulski, R.J.3
-
34
-
-
0000609138
-
Adeno-associated viral vectors
-
Friedman T (ed.). Cold Spring Harbor Laboratory Press: Cold Spring Harbor, NY
-
Samulski RJ, Sally M, Muzyczka N. Adeno-associated viral vectors. In The Development of Human Gene Therapy, Friedman T (ed.). Cold Spring Harbor Laboratory Press: Cold Spring Harbor, NY, 1999; 131-172.
-
(1999)
The Development of Human Gene Therapy
, pp. 131-172
-
-
Samulski, R.J.1
Sally, M.2
Muzyczka, N.3
-
35
-
-
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
-
36
-
-
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
-
37
-
-
0030902985
-
Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus
-
Herzog RW, Hagstrom JN, Kung SH, et al. Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus. Proc Natl Acad Sci USA 1997; 94: 5804-5809.
-
(1997)
Proc. Natl. Acad. Sci. USA
, vol.94
, pp. 5804-5809
-
-
Herzog, R.W.1
Hagstrom, J.N.2
Kung, S.H.3
-
38
-
-
0034517418
-
Longterm gene transfer to mouse fetuses with recombinant adenovirus and adeno-associated virus (AAV) vectors
-
Mitchell M, Jerebtsova M, Batshaw ML, Newman K, Ye X. Longterm gene transfer to mouse fetuses with recombinant adenovirus and adeno-associated virus (AAV) vectors. Gene Ther 2000; 7: 1986-1992.
-
(2000)
Gene Ther.
, vol.7
, pp. 1986-1992
-
-
Mitchell, M.1
Jerebtsova, M.2
Batshaw, M.L.3
Newman, K.4
Ye, X.5
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