-
1
-
-
77949343035
-
The effect of local over-expression of VEGF on the uterine arteries of pregnant sheep
-
Abi-Nader K, Mehta V, Torondel B, et al. 2009a. The effect of local over-expression of VEGF on the uterine arteries of pregnant sheep. Reprod Sci 16: 77A.
-
(2009)
Reprod Sci
, vol.16
-
-
Abi-Nader, K.1
Mehta, V.2
Torondel, B.3
-
3
-
-
0036022679
-
Anticholinergic suppression of fetal rabbit upper gastrointestinal motility
-
Acosta R, Lee JJ, Oyachi N, Buchmiller-Crair TL, Atkinson JB, Ross MG. 2002. Anticholinergic suppression of fetal rabbit upper gastrointestinal motility. J Matern Fetal Neonatal Med 11: 153-157.
-
(2002)
J Matern Fetal Neonatal Med
, vol.11
, pp. 153-157
-
-
Acosta, R.1
Lee, J.J.2
Oyachi, N.3
Buchmiller-Crair, T.L.4
Atkinson, J.B.5
Ross, M.G.6
-
4
-
-
0034641893
-
Mdx mice inducibly expressing dystrophin provide insights into the potential of gene therapy for Duchenne muscular dystrophy
-
Ahmad A, Brinson M, Hodges BL, Chamberlain JS, Amalfitano A. 2000. Mdx mice inducibly expressing dystrophin provide insights into the potential of gene therapy for Duchenne muscular dystrophy. Hum Mol Genet 9: 2507-2515.
-
(2000)
Hum Mol Genet
, vol.9
, pp. 2507-2515
-
-
Ahmad, A.1
Brinson, M.2
Hodges, B.L.3
Chamberlain, J.S.4
Amalfitano, A.5
-
5
-
-
12444283327
-
Transgenes encompassing dual-promoter CpG islands from the human TBP and HNRPA2B1 loci are resistant to heterochromatin-mediated silencing
-
Antoniou M, Harland L, Mustoe T, et al. 2003. Transgenes encompassing dual-promoter CpG islands from the human TBP and HNRPA2B1 loci are resistant to heterochromatin-mediated silencing. Genomics 82: 269-279.
-
(2003)
Genomics
, vol.82
, pp. 269-279
-
-
Antoniou, M.1
Harland, L.2
Mustoe, T.3
-
6
-
-
33746865345
-
In vivo delivery of recombinant viruses to the fetal murine cochlea: transduction characteristics and long-term effects on auditory function
-
Bedrosian JC, Gratton MA, Brigande JV, Tang W, Landau J, Bennett J. 2006. In vivo delivery of recombinant viruses to the fetal murine cochlea: transduction characteristics and long-term effects on auditory function. Mol Ther 14: 328-335.
-
(2006)
Mol Ther
, vol.14
, pp. 328-335
-
-
Bedrosian, J.C.1
Gratton, M.A.2
Brigande, J.V.3
Tang, W.4
Landau, J.5
Bennett, J.6
-
7
-
-
20844454532
-
Patterns of gene expression from in utero delivery of adenoviral-associated vector serotype 1
-
Bilbao R, Reay DP, Li J, Xiao X, Clemens PR. 2005. Patterns of gene expression from in utero delivery of adenoviral-associated vector serotype 1. Hum Gene Ther 16: 678-684.
-
(2005)
Hum Gene Ther
, vol.16
, pp. 678-684
-
-
Bilbao, R.1
Reay, D.P.2
Li, J.3
Xiao, X.4
Clemens, P.R.5
-
8
-
-
0005984244
-
Actively acquired tolerance of foreign cells
-
Billingham RE, Brent L, Medawar PB. 1953. Actively acquired tolerance of foreign cells. Nature 172: 603-606.
-
(1953)
Nature
, vol.172
, pp. 603-606
-
-
Billingham, R.E.1
Brent, L.2
Medawar, P.B.3
-
9
-
-
0001642715
-
Quantitative studies on tissue transplantation immunity III actively acquired tolerance
-
Billingham RE, Brent L, Medawar PB. 1956. Quantitative studies on tissue transplantation immunity III actively acquired tolerance. Phil Transact R Soc Biol Sci (Lond) B239: 357-369.
-
(1956)
Phil Transact R Soc Biol Sci (Lond)
, vol.B239
, pp. 357-369
-
-
Billingham, R.E.1
Brent, L.2
Medawar, P.B.3
-
10
-
-
79955012379
-
In vitro and in vivo cardiomyogenic differentiation of amniotic fluid stem cells
-
Bollini S, Pozzobon M, Nobles M, et al. 2010. In vitro and in vivo cardiomyogenic differentiation of amniotic fluid stem cells. Stem Cell Rev 7(2): 364-380.
-
(2010)
Stem Cell Rev
, vol.7
, Issue.2
, pp. 364-380
-
-
Bollini, S.1
Pozzobon, M.2
Nobles, M.3
-
11
-
-
3242734178
-
Long-term transgene expression in cardiac and skeletal muscle following fetal administration of adenoviral or adeno-associated viral vectors in mice
-
Bouchard S, MacKenzie TC, Radu AP, et al. 2003. Long-term transgene expression in cardiac and skeletal muscle following fetal administration of adenoviral or adeno-associated viral vectors in mice. J Gene Med 5: 941-950.
-
(2003)
J Gene Med
, vol.5
, pp. 941-950
-
-
Bouchard, S.1
MacKenzie, T.C.2
Radu, A.P.3
-
12
-
-
0022913087
-
Prenatal diagnosis in 200 pregnancies with a 1-in-4 risk of cystic fibrosis
-
Boué A, Muller F, Nezelof C, et al. 1986. Prenatal diagnosis in 200 pregnancies with a 1-in-4 risk of cystic fibrosis. Hum Genet 74: 288-297.
-
(1986)
Hum Genet
, vol.74
, pp. 288-297
-
-
Boué, A.1
Muller, F.2
Nezelof, C.3
-
13
-
-
0034893257
-
In utero AAV-mediated gene transfer to rabbit pulmonary epithelium
-
Boyle MP, Enke RA, Adams RJ, Guggino WB, Zeitlin PL. 2001. In utero AAV-mediated gene transfer to rabbit pulmonary epithelium. Mol Ther 4: 115-121.
-
(2001)
Mol Ther
, vol.4
, pp. 115-121
-
-
Boyle, M.P.1
Enke, R.A.2
Adams, R.J.3
Guggino, W.B.4
Zeitlin, P.L.5
-
14
-
-
36849053006
-
A microRNA-regulated lentiviral vector mediates stable correction of hemophilia B mice
-
Brown BD, Cantore A, Annoni A, et al. 2007a. A microRNA-regulated lentiviral vector mediates stable correction of hemophilia B mice. Blood 110(13): 4144-4152.
-
(2007)
Blood
, vol.110
, Issue.13
, pp. 4144-4152
-
-
Brown, B.D.1
Cantore, A.2
Annoni, A.3
-
15
-
-
36849060584
-
Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state
-
Brown BD, Gentner B, Cantore A, et al. 2007b. Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state. Nat Biotechnol 25(12): 1457-1467.
-
(2007)
Nat Biotechnol
, vol.25
, Issue.12
, pp. 1457-1467
-
-
Brown, B.D.1
Gentner, B.2
Cantore, A.3
-
16
-
-
23844497594
-
Factors influencing adenovirus mediated airway transduction in fetal mice
-
Buckley SMK. 2005. Factors influencing adenovirus mediated airway transduction in fetal mice. Mol Ther 12(3): 484-492.
-
(2005)
Mol Ther
, vol.12
, Issue.3
, pp. 484-492
-
-
Buckley, S.M.K.1
-
17
-
-
42549089142
-
Intra-amniotic application of CFTR-expressing adenovirus does not reverse cystic fibrosis phenotype in inbred Cftr-knockout mice
-
Buckley SMK, Waddington SN, Jezzard S, et al. 2008. Intra-amniotic application of CFTR-expressing adenovirus does not reverse cystic fibrosis phenotype in inbred Cftr-knockout mice. Mol Ther 16: 819-824.
-
(2008)
Mol Ther
, vol.16
, pp. 819-824
-
-
Buckley, S.M.K.1
Waddington, S.N.2
Jezzard, S.3
-
18
-
-
77956928344
-
Transfusion independence and HMGA2 activation after gene therapy of human beta-thalassaemia
-
Cavazzana-Calvo M, Payen E, Negre O, et al. 2010. Transfusion independence and HMGA2 activation after gene therapy of human beta-thalassaemia. Nature 467(7313): 318-322.
-
(2010)
Nature
, vol.467
, Issue.7313
, pp. 318-322
-
-
Cavazzana-Calvo, M.1
Payen, E.2
Negre, O.3
-
19
-
-
0042526080
-
Gene therapy progress and prospects: gene therapy of lysosomal storage disorders
-
Cheng SH, Smith AE. 2003. Gene therapy progress and prospects: gene therapy of lysosomal storage disorders. Gene Ther 10: 1275-1281.
-
(2003)
Gene Ther
, vol.10
, pp. 1275-1281
-
-
Cheng, S.H.1
Smith, A.E.2
-
20
-
-
52449135543
-
Early fetal gene delivery utilizes both central and peripheral mechanisms of tolerance induction
-
Colletti E, Lindstedt S, Park PJ, Almeida-Porada G, Porada CD. 2008. Early fetal gene delivery utilizes both central and peripheral mechanisms of tolerance induction. Exp Hematol 36: 816-822.
-
(2008)
Exp Hematol
, vol.36
, pp. 816-822
-
-
Colletti, E.1
Lindstedt, S.2
Park, P.J.3
Almeida-Porada, G.4
Porada, C.D.5
-
21
-
-
0037338159
-
Ultrasound guided percutaneous delivery of adenoviral vectors encoding the beta-galactosidase and human factor IX genes to early gestation fetal sheep in utero
-
David AL, Cook T, Waddington S, et al. 2003a. Ultrasound guided percutaneous delivery of adenoviral vectors encoding the beta-galactosidase and human factor IX genes to early gestation fetal sheep in utero. Hum Gene Ther 14: 353-364.
-
(2003)
Hum Gene Ther
, vol.14
, pp. 353-364
-
-
David, A.L.1
Cook, T.2
Waddington, S.3
-
22
-
-
0043163482
-
Percutaneous ultrasound-guided injection of the trachea in fetal sheep: a novel technique to target the fetal airways
-
David AL, Peebles DM, Gregory L, et al. 2003b. Percutaneous ultrasound-guided injection of the trachea in fetal sheep: a novel technique to target the fetal airways. Fetal Diagn Ther 18: 385-390.
-
(2003)
Fetal Diagn Ther
, vol.18
, pp. 385-390
-
-
David, A.L.1
Peebles, D.M.2
Gregory, L.3
-
23
-
-
33746218856
-
Clinically applicable procedure for gene delivery to fetal gut by ultrasound-guided gastric injection: toward prenatal prevention of early-onset intestinal diseases
-
David AL, Peebles DM, Gregory L, et al. 2006a. Clinically applicable procedure for gene delivery to fetal gut by ultrasound-guided gastric injection: toward prenatal prevention of early-onset intestinal diseases. Hum Gene Ther 17: 767-779.
-
(2006)
Hum Gene Ther
, vol.17
, pp. 767-779
-
-
David, A.L.1
Peebles, D.M.2
Gregory, L.3
-
24
-
-
33746558043
-
Ultrasound-guided injection and occlusion of the trachea in fetal sheep
-
David AL, Weisz B, Gregory L, et al. 2006b. Ultrasound-guided injection and occlusion of the trachea in fetal sheep. Ultrasound Obstet Gynaecol 28: 82-88.
-
(2006)
Ultrasound Obstet Gynaecol
, vol.28
, pp. 82-88
-
-
David, A.L.1
Weisz, B.2
Gregory, L.3
-
25
-
-
52049125232
-
Local delivery of VEGF adenovirus to the uterine artery increases vasorelaxation and uterine blood flow in the pregnant sheep
-
David AL, Torondel B, Zachary I, et al. 2008. Local delivery of VEGF adenovirus to the uterine artery increases vasorelaxation and uterine blood flow in the pregnant sheep. Gene Ther 15: 1344-1350.
-
(2008)
Gene Ther
, vol.15
, pp. 1344-1350
-
-
David, A.L.1
Torondel, B.2
Zachary, I.3
-
26
-
-
77953240664
-
Ultrasonographic development of the fetal sheep stomach and evaluation of early gestation ultrasound-guided in utero intragastric injection
-
David AL, Abi-Nader KN, Weisz B, et al. 2010. Ultrasonographic development of the fetal sheep stomach and evaluation of early gestation ultrasound-guided in utero intragastric injection. Taiwan J Obstet Gynecol 49: 23-29.
-
(2010)
Taiwan J Obstet Gynecol
, vol.49
, pp. 23-29
-
-
David, A.L.1
Abi-Nader, K.N.2
Weisz, B.3
-
27
-
-
79954543052
-
rAAV mediated in utero gene transfer gives therapeutic transgene expression in the sheep
-
David AL, McIntosh J, Peebles DM, et al. 2011. rAAV mediated in utero gene transfer gives therapeutic transgene expression in the sheep. Hum Gene Ther 22(4): 419-426.
-
(2011)
Hum Gene Ther
, vol.22
, Issue.4
, pp. 419-426
-
-
David, A.L.1
McIntosh, J.2
Peebles, D.M.3
-
28
-
-
42549129355
-
Adenovirus-mediated in utero expressin of CFTR does not improve survival of CFTR knockout mice
-
Davies SA, Varathalingam A, Painter H, et al. 2008. Adenovirus-mediated in utero expressin of CFTR does not improve survival of CFTR knockout mice. Mol Ther 16(5): 812-818.
-
(2008)
Mol Ther
, vol.16
, Issue.5
, pp. 812-818
-
-
Davies, S.A.1
Varathalingam, A.2
Painter, H.3
-
29
-
-
33846120651
-
Isolation of amniotic stem cell lines with potential for therapy
-
De Coppi P, Bartsch G Jr, Siddiqui MM, et al. 2007. Isolation of amniotic stem cell lines with potential for therapy. Nat Biotechnol 25: 100.
-
(2007)
Nat Biotechnol
, vol.25
, pp. 100
-
-
De Coppi, P.1
Bartsch Jr, G.2
Siddiqui, M.M.3
-
30
-
-
10744230959
-
In utero gene therapy rescues vision in a murine model of congenital blindness
-
Dejneka NS, Surace EM, Aleman TS, et al. 2004. In utero gene therapy rescues vision in a murine model of congenital blindness. Mol Ther 9: 182-188.
-
(2004)
Mol Ther
, vol.9
, pp. 182-188
-
-
Dejneka, N.S.1
Surace, E.M.2
Aleman, T.S.3
-
31
-
-
0141594934
-
Advances in Duchenne muscular dystrophy gene therapy
-
van Deutekom JC, van Ommen GJ. 2003. Advances in Duchenne muscular dystrophy gene therapy. Nat Rev Genet 4(10): 774-783.
-
(2003)
Nat Rev Genet
, vol.4
, Issue.10
, pp. 774-783
-
-
van Deutekom, J.C.1
van Ommen, G.J.2
-
32
-
-
65449170084
-
Human and murine amniotic fluid c-Kit + Lin- cells display hematopoietic activity
-
Ditadi A, De Coppi P, Picone O, et al. 2009. Human and murine amniotic fluid c-Kit + Lin- cells display hematopoietic activity. Blood 113: 3953-3960.
-
(2009)
Blood
, vol.113
, pp. 3953-3960
-
-
Ditadi, A.1
De Coppi, P.2
Picone, O.3
-
33
-
-
0029793791
-
Effect of amniotic fluid on cationic lipid mediated transfection and viral infection
-
Douar A-M, Themis M, Sandig V, Friedmann T, Coutelle C. 1996. Effect of amniotic fluid on cationic lipid mediated transfection and viral infection. Gene Ther 3: 789-796.
-
(1996)
Gene Ther
, vol.3
, pp. 789-796
-
-
Douar, A.-M.1
Themis, M.2
Sandig, V.3
Friedmann, T.4
Coutelle, C.5
-
34
-
-
0030867444
-
Foetal gene delivery in mice by intra-amniotic administration of retroviral producer cells and adenovirus
-
Douar A-M, Adebakin S, Themis M, Pavirani A, Cook T, Coutelle C. 1997. Foetal gene delivery in mice by intra-amniotic administration of retroviral producer cells and adenovirus. Gene Ther 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
-
35
-
-
2542568707
-
Distal intestinal obstruction syndrome in adults with cystic fibrosis
-
Dray X, Bienvenu T, Desmazes-Dufeu N, Dusser D, Marteau P, Hubert D. 2004. Distal intestinal obstruction syndrome in adults with cystic fibrosis. Clin Gastroenterol Hepatol 2: 498-503.
-
(2004)
Clin Gastroenterol Hepatol
, vol.2
, pp. 498-503
-
-
Dray, X.1
Bienvenu, T.2
Desmazes-Dufeu, N.3
Dusser, D.4
Marteau, P.5
Hubert, D.6
-
36
-
-
0026687233
-
- channels by protein kinase A corrected by insertion of CFTR
-
- channels by protein kinase A corrected by insertion of CFTR. Nature 358: 581-584.
-
(1992)
Nature
, vol.358
, pp. 581-584
-
-
Egan, M.1
Flotte, T.2
Afione, S.3
-
37
-
-
33847200820
-
Gene transfer to ocular stem cells by early gestational intraamniotic injection of lentiviral vector
-
Endo M. 2007. Gene transfer to ocular stem cells by early gestational intraamniotic injection of lentiviral vector. Mol Ther 15: 579-587.
-
(2007)
Mol Ther
, vol.15
, pp. 579-587
-
-
Endo, M.1
-
38
-
-
37549055397
-
Efficient in vivo targeting of epidermal stem cells by early gestational intraamniotic injection of lentiviral vector driven by the keratin 5 promoter
-
Endo M, Zoltick PW, Peranteau WH, et al. 2008. Efficient in vivo targeting of epidermal stem cells by early gestational intraamniotic injection of lentiviral vector driven by the keratin 5 promoter. Mol Ther 16: 131-137.
-
(2008)
Mol Ther
, vol.16
, pp. 131-137
-
-
Endo, M.1
Zoltick, P.W.2
Peranteau, W.H.3
-
39
-
-
74349131596
-
The developmental stage determines the distribution and duration of gene expression after early intra-amniotic gene transfer using lentiviral vectors
-
Endo M, Henriques-Coelho T, Zoltick PW, et al. 2010. The developmental stage determines the distribution and duration of gene expression after early intra-amniotic gene transfer using lentiviral vectors. Gene Ther 17: 61-71.
-
(2010)
Gene Ther
, vol.17
, pp. 61-71
-
-
Endo, M.1
Henriques-Coelho, T.2
Zoltick, P.W.3
-
40
-
-
0036113958
-
Fetal gene transfer by intrauterine injection with microbubble-enhanced ultrasound
-
Endoh M, Koibuchi N, Sato M, et al. 2002. Fetal gene transfer by intrauterine injection with microbubble-enhanced ultrasound. Mol Ther 5: 501-508.
-
(2002)
Mol Ther
, vol.5
, pp. 501-508
-
-
Endoh, M.1
Koibuchi, N.2
Sato, M.3
-
41
-
-
0034882342
-
Targeted gene transfer to lymphocytes using murine leukaemia virus vectors pseudotyped with spleen necrosis virus envelope proteins
-
Engelstädter M, Buchholz CJ, Bobkova M, et al. 2001. Targeted gene transfer to lymphocytes using murine leukaemia virus vectors pseudotyped with spleen necrosis virus envelope proteins. Gene Ther 8: 1202-1206.
-
(2001)
Gene Ther
, vol.8
, pp. 1202-1206
-
-
Engelstädter, M.1
Buchholz, C.J.2
Bobkova, M.3
-
42
-
-
60149106907
-
Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes
-
Foust KD, Nurre E, Montgomery CL, Hernandez A, Chan CM, Kaspar BK. 2009. Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes. Nat Biotechnol 27: 59-65.
-
(2009)
Nat Biotechnol
, vol.27
, pp. 59-65
-
-
Foust, K.D.1
Nurre, E.2
Montgomery, C.L.3
Hernandez, A.4
Chan, C.M.5
Kaspar, B.K.6
-
43
-
-
77749249680
-
Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN
-
Foust KD, Wang X, McGovern VL, et al. 2010. Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN. Nat Biotechnol 28: 271-274.
-
(2010)
Nat Biotechnol
, vol.28
, pp. 271-274
-
-
Foust, K.D.1
Wang, X.2
McGovern, V.L.3
-
44
-
-
1142298437
-
The peritoneal route as a safe pathway for early in utero therapies: illustration by a 12-year follow-up after conservative management of severe Rhesus allo-immunization
-
Gallot D, Boiret N, Vanlieferinghen P, et al. 2004. The peritoneal route as a safe pathway for early in utero therapies: illustration by a 12-year follow-up after conservative management of severe Rhesus allo-immunization. Fetal Diagn Ther 19: 170-173.
-
(2004)
Fetal Diagn Ther
, vol.19
, pp. 170-173
-
-
Gallot, D.1
Boiret, N.2
Vanlieferinghen, P.3
-
45
-
-
3042609692
-
In utero recombinant adeno-associated virus gene transfer in mice, rats, and primates
-
Garrett DJ, Larson JE, Dunn D, Marrero L, Cohen JC. 2003. In utero recombinant adeno-associated virus gene transfer in mice, rats, and primates. BMC Biotechnol 3: 16.
-
(2003)
BMC Biotechnol
, vol.3
, pp. 16
-
-
Garrett, D.J.1
Larson, J.E.2
Dunn, D.3
Marrero, L.4
Cohen, J.C.5
-
46
-
-
51249102804
-
Cystic adenomatoid malformations are induced by localized FGF10 overexpression in fetal rat lung
-
Gonzaga S, Henriques-Coelho T, Davey M, et al. 2008. Cystic adenomatoid malformations are induced by localized FGF10 overexpression in fetal rat lung. Am J Respir Cell Mol Biol 39(3): 346-355.
-
(2008)
Am J Respir Cell Mol Biol
, vol.39
, Issue.3
, pp. 346-355
-
-
Gonzaga, S.1
Henriques-Coelho, T.2
Davey, M.3
-
47
-
-
0037222237
-
Enhancement of adenovirus-mediated gene transfer to the airways by DEAE dextran and sodium caprate in vivo
-
Gregory LG, Harbottle RP, Lawrence L, Knapton HJ, Themis M, Coutelle C. 2002. Enhancement of adenovirus-mediated gene transfer to the airways by DEAE dextran and sodium caprate in vivo. Mol Ther 7: 19-26.
-
(2002)
Mol Ther
, vol.7
, pp. 19-26
-
-
Gregory, L.G.1
Harbottle, R.P.2
Lawrence, L.3
Knapton, H.J.4
Themis, M.5
Coutelle, C.6
-
48
-
-
4243132862
-
Highly efficient EIAV-mediated in utero gene transfer and expression in the major muscle groups affected by Duchenne muscular dystrophy
-
Gregory LG, Waddington SN, Holder MV, et al. 2004. Highly efficient EIAV-mediated in utero gene transfer and expression in the major muscle groups affected by Duchenne muscular dystrophy. Gene Ther 11: 1117-1125.
-
(2004)
Gene Ther
, vol.11
, pp. 1117-1125
-
-
Gregory, L.G.1
Waddington, S.N.2
Holder, M.V.3
-
49
-
-
52949112282
-
Functional auditory hair cells produced in the mammalian cochlea by in utero gene transfer
-
Gubbels SP, Woessner DW, Mitchell JC, Ricci AJ, Brigande JV. 2008. Functional auditory hair cells produced in the mammalian cochlea by in utero gene transfer. Nature 455(7212): 537-541.
-
(2008)
Nature
, vol.455
, Issue.7212
, pp. 537-541
-
-
Gubbels, S.P.1
Woessner, D.W.2
Mitchell, J.C.3
Ricci, A.J.4
Brigande, J.V.5
-
50
-
-
50549093286
-
Gene transfer: the challenge of regulated gene expression
-
Guo ZS, Li Q, Bartlett DL, Yang JY, Fang B. 2008. Gene transfer: the challenge of regulated gene expression. Trends Mol Med 14(9): 410-418.
-
(2008)
Trends Mol Med
, vol.14
, Issue.9
, pp. 410-418
-
-
Guo, Z.S.1
Li, Q.2
Bartlett, D.L.3
Yang, J.Y.4
Fang, B.5
-
51
-
-
34547416185
-
Fetal gene therapy of α-thalassemia in a mouse model
-
Han XD, Lin C, Chang J, Sadelain M, Kan YW. 2007. Fetal gene therapy of α-thalassemia in a mouse model. PNAS 104: 9007-9011.
-
(2007)
PNAS
, vol.104
, pp. 9007-9011
-
-
Han, X.D.1
Lin, C.2
Chang, J.3
Sadelain, M.4
Kan, Y.W.5
-
52
-
-
33846272370
-
Targeted gene transfer to fetal rat lung interstitium by ultrasound-guided intrapulmonary injection
-
Henriques-Coelho T. 2007. Targeted gene transfer to fetal rat lung interstitium by ultrasound-guided intrapulmonary injection. Mol Ther 15: 340-347.
-
(2007)
Mol Ther
, vol.15
, pp. 340-347
-
-
Henriques-Coelho, T.1
-
53
-
-
0028803618
-
Intraamniotic administration of an adenoviral vector for gene transfer to fetal sheep and mouse tissues
-
Holzinger A, Trapnell BC, Weaver TE, Whitsett JA, Iwamoto HS. 1995. Intraamniotic administration of an adenoviral vector for gene transfer to fetal sheep and mouse tissues. Pediatr Res 38: 844-850.
-
(1995)
Pediatr Res
, vol.38
, pp. 844-850
-
-
Holzinger, A.1
Trapnell, B.C.2
Weaver, T.E.3
Whitsett, J.A.4
Iwamoto, H.S.5
-
54
-
-
34548102639
-
Functional analysis of various promoters in lentiviral vectors at different stages of in vitro differentiation of mouse embryonic stem cells
-
Hong S, Hwang DY, Yoon S, et al. 2007. Functional analysis of various promoters in lentiviral vectors at different stages of in vitro differentiation of mouse embryonic stem cells. Mol Ther 15(9): 1630-1639.
-
(2007)
Mol Ther
, vol.15
, Issue.9
, pp. 1630-1639
-
-
Hong, S.1
Hwang, D.Y.2
Yoon, S.3
-
55
-
-
51349158298
-
Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients
-
Howe SJ, Mansour MR, Schwarzwaelder K, et al. 2008. Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients. J Clin Investig 118: 3143-3150.
-
(2008)
J Clin Investig
, vol.118
, pp. 3143-3150
-
-
Howe, S.J.1
Mansour, M.R.2
Schwarzwaelder, K.3
-
56
-
-
0029584267
-
Differential short-term transduction efficiency of adult versus newborn mouse tissues by adenoviral recombinants
-
Huard J, Lochmuller H, Acsadi G, et al. 1995. Differential short-term transduction efficiency of adult versus newborn mouse tissues by adenoviral recombinants. Exp Mol Pathol 62(2): 131-143.
-
(1995)
Exp Mol Pathol
, vol.62
, Issue.2
, pp. 131-143
-
-
Huard, J.1
Lochmuller, H.2
Acsadi, G.3
-
57
-
-
69949111663
-
Severe diaphragmatic hernia treated by fetal endoscopic tracheal occlusion
-
Jani JC, Nicolaides KH, Gratacós E, et al. 2009. Severe diaphragmatic hernia treated by fetal endoscopic tracheal occlusion. Ultrasound Obstet Gynecol 34: 304-310.
-
(2009)
Ultrasound Obstet Gynecol
, vol.34
, pp. 304-310
-
-
Jani, J.C.1
Nicolaides, K.H.2
Gratacós, E.3
-
58
-
-
0026918861
-
Efficiency of gene transfer for restoration of normal airway epithelial function in cystic fibrosis
-
Johnson LG, Olsen JC, Sarkadi B, Moore KL, Swanstrom R, Boucher RC. 1992. Efficiency of gene transfer for restoration of normal airway epithelial function in cystic fibrosis. Nat Genet 2: 21-25.
-
(1992)
Nat Genet
, vol.2
, pp. 21-25
-
-
Johnson, L.G.1
Olsen, J.C.2
Sarkadi, B.3
Moore, K.L.4
Swanstrom, R.5
Boucher, R.C.6
-
59
-
-
0035105820
-
Filovirus-pseudotyped lentiviral vector can efficiently and stably transduce airway epithelia in vivo
-
Kobinger GP, Weiner DJ, Yu QC, Wilson JM. 2001. Filovirus-pseudotyped lentiviral vector can efficiently and stably transduce airway epithelia in vivo. Nat Biotechnol 19: 225-230.
-
(2001)
Nat Biotechnol
, vol.19
, pp. 225-230
-
-
Kobinger, G.P.1
Weiner, D.J.2
Yu, Q.C.3
Wilson, J.M.4
-
60
-
-
70249117075
-
Systemic delivery of AAV8 in utero results in gene expression in diaphragm and limb muscle: treatment implications for muscle disorders
-
Koppanati BM, Li J, Xiao X, Clemens PR. 2009. Systemic delivery of AAV8 in utero results in gene expression in diaphragm and limb muscle: treatment implications for muscle disorders. Gene Ther 16: 1130-1137.
-
(2009)
Gene Ther
, vol.16
, pp. 1130-1137
-
-
Koppanati, B.M.1
Li, J.2
Xiao, X.3
Clemens, P.R.4
-
61
-
-
78149469593
-
Improvement of the mdx mouse dystrophic phenotype by systemic in utero AAV8 delivery of a minidystrophin gene
-
Koppanati BM, Li J, Reay DP, et al. 2010. Improvement of the mdx mouse dystrophic phenotype by systemic in utero AAV8 delivery of a minidystrophin gene. Gene Ther 17(11): 1355-1362.
-
(2010)
Gene Ther
, vol.17
, Issue.11
, pp. 1355-1362
-
-
Koppanati, B.M.1
Li, J.2
Reay, D.P.3
-
62
-
-
0036447246
-
A preliminary evaluation of recombinant adeno-associated virus biodistribution in rhesus monkeys after intrahepatic inoculation in utero
-
Lai L, Davison BB, Veazey RS, Fisher KJ, Baskin GB. 2002. A preliminary evaluation of recombinant adeno-associated virus biodistribution in rhesus monkeys after intrahepatic inoculation in utero. Hum Gene Ther 13: 2027-2039.
-
(2002)
Hum Gene Ther
, vol.13
, pp. 2027-2039
-
-
Lai, L.1
Davison, B.B.2
Veazey, R.S.3
Fisher, K.J.4
Baskin, G.B.5
-
63
-
-
33745686074
-
Improvement of pulmonary hypoplasia associated with congenital diaphragmatic hernia by in utero CFTR gene therapy
-
Larson JE, Cohen CJ. 2006. Improvement of pulmonary hypoplasia associated with congenital diaphragmatic hernia by in utero CFTR gene therapy. Am J Physiol Lung Cell Mol Physiol 291: L4-L10.
-
(2006)
Am J Physiol Lung Cell Mol Physiol
, vol.291
-
-
Larson, J.E.1
Cohen, C.J.2
-
64
-
-
0031040502
-
Reversal of cystic fibrosis phenotype in mice by gene therapy in utero
-
Larson JE, Morrow SL, Happel L, Sharp JF, Cohen JC. 1997. Reversal of cystic fibrosis phenotype in mice by gene therapy in utero. Lancet 349: 619-620.
-
(1997)
Lancet
, vol.349
, pp. 619-620
-
-
Larson, J.E.1
Morrow, S.L.2
Happel, L.3
Sharp, J.F.4
Cohen, J.C.5
-
65
-
-
0034577051
-
Gene transfer into the fetal primate: evidence for the secretion of transgene product
-
Larson JE, Morrow SL, Delcarpio JB, et al. 2000. Gene transfer into the fetal primate: evidence for the secretion of transgene product. Mol Ther 2: 631-639.
-
(2000)
Mol Ther
, vol.2
, pp. 631-639
-
-
Larson, J.E.1
Morrow, S.L.2
Delcarpio, J.B.3
-
66
-
-
18444399007
-
Fetal gene transfer using lentiviral vectors and the potential for germ cell transduction in rhesus monkeys (Macaca mulatta)
-
Lee CC, Jimenez DF, Kohn DB, Tarantal AF. 2005. Fetal gene transfer using lentiviral vectors and the potential for germ cell transduction in rhesus monkeys (Macaca mulatta). Hum Gene Ther 16: 417-425.
-
(2005)
Hum Gene Ther
, vol.16
, pp. 417-425
-
-
Lee, C.C.1
Jimenez, D.F.2
Kohn, D.B.3
Tarantal, A.F.4
-
67
-
-
77954036603
-
Placenta as a newly identified source of hematopoietic stem cells
-
Lee LK, Ueno M, Van Handel B, Mikkola HK. 2010. Placenta as a newly identified source of hematopoietic stem cells. Curr Opin Hematol 17(4): 313-318.
-
(2010)
Curr Opin Hematol
, vol.17
, Issue.4
, pp. 313-318
-
-
Lee, L.K.1
Ueno, M.2
Van Handel, B.3
Mikkola, H.K.4
-
68
-
-
0034981261
-
In utero delivery of adeno-associated viral vectors: intraperitoneal gene transfer produces long-term expression
-
Lipshutz GS, Gruber CA, Cao Y, Hardy J, Contag CH, Gaensler KML. 2001. In utero delivery of adeno-associated viral vectors: intraperitoneal gene transfer produces long-term expression. Mol Ther 3: 284-292.
-
(2001)
Mol Ther
, vol.3
, pp. 284-292
-
-
Lipshutz, G.S.1
Gruber, C.A.2
Cao, Y.3
Hardy, J.4
Contag, C.H.5
Gaensler, K.M.L.6
-
69
-
-
12744274695
-
Transduction of satellite cells after prenatal intramuscular administration of lentiviral vectors
-
MacKenzie TC, Kobinger GP, Louboutin JP, et al. 2005. Transduction of satellite cells after prenatal intramuscular administration of lentiviral vectors. J Gene Med 7: 50-58.
-
(2005)
J Gene Med
, vol.7
, pp. 50-58
-
-
MacKenzie, T.C.1
Kobinger, G.P.2
Louboutin, J.P.3
-
70
-
-
0033506343
-
Neonatal onset ornithine transcarbamylase deficiency: a retrospective analysis
-
Maestri NE, Clissold D, Brusilow SW. 1999. Neonatal onset ornithine transcarbamylase deficiency: a retrospective analysis. J Pediatr 134: 268-272.
-
(1999)
J Pediatr
, vol.134
, pp. 268-272
-
-
Maestri, N.E.1
Clissold, D.2
Brusilow, S.W.3
-
71
-
-
80455174043
-
Stable human FIX expression after 0.9G intrauterine gene transfer of self-complementary adeno-associated viral vector 5 and 8 in macaques
-
accepted).
-
Mattar CN, Nathwani AC, Waddington SN, et al. 2011. Stable human FIX expression after 0.9G intrauterine gene transfer of self-complementary adeno-associated viral vector 5 and 8 in macaques. Mol Ther (accepted).
-
(2011)
Mol Ther
-
-
Mattar, C.N.1
Nathwani, A.C.2
Waddington, S.N.3
-
72
-
-
0035165403
-
Factor VII deficiency and the FVII mutation database
-
McVey JH, Boswell E, Mumford AD, Kemball-Cook G, Tuddenham EG. 2001. Factor VII deficiency and the FVII mutation database. Hum Mutat 17: 3-17.
-
(2001)
Hum Mutat
, vol.17
, pp. 3-17
-
-
McVey, J.H.1
Boswell, E.2
Mumford, A.D.3
Kemball-Cook, G.4
Tuddenham, E.G.5
-
73
-
-
0036412405
-
In utero injection of alpha-L-iduronidase-carrying retrovirus in canine mucopolysaccharidosis type I: infection of multiple tissues and neonatal gene expression
-
Meertens L, Zhao Y, Rosic-Kablar S, et al. 2002. In utero injection of alpha-L-iduronidase-carrying retrovirus in canine mucopolysaccharidosis type I: infection of multiple tissues and neonatal gene expression. Hum Gene Ther 13: 1809-1820.
-
(2002)
Hum Gene Ther
, vol.13
, pp. 1809-1820
-
-
Meertens, L.1
Zhao, Y.2
Rosic-Kablar, S.3
-
75
-
-
4444333453
-
Long-term correction of ornithine transcarbamylase deficiency by WPRE-mediated overexpression using a helper-dependent adenovirus
-
Mian A, McCormack WM Jr, Mane V, et al. 2004. Long-term correction of ornithine transcarbamylase deficiency by WPRE-mediated overexpression using a helper-dependent adenovirus. Mol Ther 10: 492-499.
-
(2004)
Mol Ther
, vol.10
, pp. 492-499
-
-
Mian, A.1
McCormack Jr, W.M.2
Mane, V.3
-
76
-
-
0034517418
-
Long-term gene transfer to mouse fetuses with recombinant adenovirus and adeno-associated virus (AAV) vectors
-
Mitchell M, Jerebtsova M, Batshaw ML, Newman K, Ye X. 2000. Long-term gene transfer to mouse fetuses with recombinant adenovirus and adeno-associated virus (AAV) vectors. Gene Ther 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
-
77
-
-
33745118671
-
Long-term correction of ammonia metabolism and prolonged survival in ornithine transcarbamylase-deficient mice following liver-directed treatment with adeno-associated viral vectors
-
Moscioni D, Morizono H, McCarter RJ, et al. 2006. Long-term correction of ammonia metabolism and prolonged survival in ornithine transcarbamylase-deficient mice following liver-directed treatment with adeno-associated viral vectors. Mol Ther 14: 25-33.
-
(2006)
Mol Ther
, vol.14
, pp. 25-33
-
-
Moscioni, D.1
Morizono, H.2
McCarter, R.J.3
-
78
-
-
0019411940
-
Stimulatory effect of theophylline on regulation of fetal breathing movements
-
Moss IR, Scarpelli EM. 1981. Stimulatory effect of theophylline on regulation of fetal breathing movements. Pediatr Res 15: 870-873.
-
(1981)
Pediatr Res
, vol.15
, pp. 870-873
-
-
Moss, I.R.1
Scarpelli, E.M.2
-
79
-
-
33751173879
-
Evaluation of prenatal intra-amniotic LAMB3 gene delivery in a mouse model of Herlitz disease
-
Muhle C, Neuner A, Park J, et al. 2006. Evaluation of prenatal intra-amniotic LAMB3 gene delivery in a mouse model of Herlitz disease. Gene Ther 13: 1665-1676.
-
(2006)
Gene Ther
, vol.13
, pp. 1665-1676
-
-
Muhle, C.1
Neuner, A.2
Park, J.3
-
80
-
-
33645528206
-
Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver
-
Nathwani AC, Gray JT, Ng CYC, et al. 2006. Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver. Blood 107: 2653-2661.
-
(2006)
Blood
, vol.107
, pp. 2653-2661
-
-
Nathwani, A.C.1
Gray, J.T.2
Ng, C.Y.C.3
-
81
-
-
0025030753
-
The risks of early cordocentesis (12-21 weeks): analysis of 500 procedures
-
Orlandi F, Damiani G, Jakil C, Lauricella S, Bertolino O, Maggio A. 1990. The risks of early cordocentesis (12-21 weeks): analysis of 500 procedures. Prenat Diagn 10: 425-428.
-
(1990)
Prenat Diagn
, vol.10
, pp. 425-428
-
-
Orlandi, F.1
Damiani, G.2
Jakil, C.3
Lauricella, S.4
Bertolino, O.5
Maggio, A.6
-
82
-
-
0035861452
-
Correction of sickle cell disease in transgenic mouse models by gene therapy
-
Pawliuk R, Westerman KA, Fabry ME, et al. 2001. Correction of sickle cell disease in transgenic mouse models by gene therapy. Science 294: 2368-2371.
-
(2001)
Science
, vol.294
, pp. 2368-2371
-
-
Pawliuk, R.1
Westerman, K.A.2
Fabry, M.E.3
-
83
-
-
9144222976
-
Widespread and efficient marker gene expression in the airway epithelia of fetal sheep after minimally invasive tracheal application of recombinant adenovirus in utero
-
Peebles D, Gregory LG, David A, et al. 2004. Widespread and efficient marker gene expression in the airway epithelia of fetal sheep after minimally invasive tracheal application of recombinant adenovirus in utero. Gene Ther 11: 70-78.
-
(2004)
Gene Ther
, vol.11
, pp. 70-78
-
-
Peebles, D.1
Gregory, L.G.2
David, A.3
-
84
-
-
0037772187
-
Successful treatment of murine β-thalassaemia using in vivo selection of genetically modified, drug-resistant hematopoietic stem cells
-
Persons DA, Allay ER, Sawai N, et al. 2003. Successful treatment of murine β-thalassaemia using in vivo selection of genetically modified, drug-resistant hematopoietic stem cells. Blood 102: 506-513.
-
(2003)
Blood
, vol.102
, pp. 506-513
-
-
Persons, D.A.1
Allay, E.R.2
Sawai, N.3
-
85
-
-
33646150228
-
Rare bleeding disorders
-
Peyvandi F, Kaufman RJ, Seligsohn U, Salomon O, Bolton-Maggs PH, Spreafico M. 2006. Rare bleeding disorders. Haemophilia 12: 137-142.
-
(2006)
Haemophilia
, vol.12
, pp. 137-142
-
-
Peyvandi, F.1
Kaufman, R.J.2
Seligsohn, U.3
Salomon, O.4
Bolton-Maggs, P.H.5
Spreafico, M.6
-
86
-
-
34447134838
-
Use of nonintegrating lentiviral vectors for gene therapy
-
Philpott NJ, Thrasher AJ. 2007. Use of nonintegrating lentiviral vectors for gene therapy. Hum Gene Ther 18: 483-489.
-
(2007)
Hum Gene Ther
, vol.18
, pp. 483-489
-
-
Philpott, N.J.1
Thrasher, A.J.2
-
87
-
-
0028314140
-
Apoptosis in human skin development: morphogenesis, periderm, and stem cells
-
Polakowska RR, Piacentini M, Bartlett R, Goldsmith LA, Haake AR. 1994. Apoptosis in human skin development: morphogenesis, periderm, and stem cells. Dev Dyn 199: 176-188.
-
(1994)
Dev Dyn
, vol.199
, pp. 176-188
-
-
Polakowska, R.R.1
Piacentini, M.2
Bartlett, R.3
Goldsmith, L.A.4
Haake, A.R.5
-
88
-
-
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 9: 1571-1585.
-
(1998)
Hum Gene Ther
, vol.9
, pp. 1571-1585
-
-
Porada, C.D.1
Tran, N.2
Eglitis, M.3
-
89
-
-
25144467369
-
Male germ-line cells are at risk following direct-injection retroviral-mediated gene transfer in utero
-
Porada CD, Park PJ, Tellez J, et al. 2005. Male germ-line cells are at risk following direct-injection retroviral-mediated gene transfer in utero. Mol Ther 12: 754-762.
-
(2005)
Mol Ther
, vol.12
, pp. 754-762
-
-
Porada, C.D.1
Park, P.J.2
Tellez, J.3
-
90
-
-
33644694447
-
Placental mesenchymal stem cells as potential autologous graft for pre- and perinatal neuroregeneration
-
Portmann-Lanz CB, Schoeberlein A, Huber A, et al. 2006. Placental mesenchymal stem cells as potential autologous graft for pre- and perinatal neuroregeneration. Am J Obstet Gynecol 194: 664-673.
-
(2006)
Am J Obstet Gynecol
, vol.194
, pp. 664-673
-
-
Portmann-Lanz, C.B.1
Schoeberlein, A.2
Huber, A.3
-
91
-
-
9444281434
-
Successful correction of the human â-thalassemia major phenotype using a lentiviral vector
-
Puthenveetil G, Scholes J, Carbonell D, et al. 2004. Successful correction of the human â-thalassemia major phenotype using a lentiviral vector. Blood 104: 3445-3453.
-
(2004)
Blood
, vol.104
, pp. 3445-3453
-
-
Puthenveetil, G.1
Scholes, J.2
Carbonell, D.3
-
92
-
-
33846005126
-
Molecular therapies in beta-thalassaemia
-
Quek L, Thein SL. 2007. Molecular therapies in beta-thalassaemia. Br J Haematol 136(3): 353-365.
-
(2007)
Br J Haematol
, vol.136
, Issue.3
, pp. 353-365
-
-
Quek, L.1
Thein, S.L.2
-
93
-
-
78649738504
-
In utero gene transfer to the mouse nervous system
-
Rahim AA, Wong AM, Buckley SM, et al. 2010a. In utero gene transfer to the mouse nervous system. Biochem Soc Trans 38: 1489-1493.
-
(2010)
Biochem Soc Trans
, vol.38
, pp. 1489-1493
-
-
Rahim, A.A.1
Wong, A.M.2
Buckley, S.M.3
-
94
-
-
78649758214
-
Efficient CNS, PNS and visceral gene delivery following fetal and intravenous administration of AAV9
-
Rahim AA, Wong AMKH, Buckley SM, et al. 2010b. Efficient CNS, PNS and visceral gene delivery following fetal and intravenous administration of AAV9. Hum Gene Ther 21: 505-506.
-
(2010)
Hum Gene Ther
, vol.21
, pp. 505-506
-
-
Rahim, A.A.1
Wong, A.M.K.H.2
Buckley, S.M.3
-
95
-
-
0036145350
-
A pilot study of in vivo liver-directed gene transfer with an adenoviral vector in partial ornithine transcarbamylase deficiency
-
Raper SE, Yudkoff M, Chirmule N, et al. 2002. A pilot study of in vivo liver-directed gene transfer with an adenoviral vector in partial ornithine transcarbamylase deficiency. Hum Gene Ther 13: 163-175.
-
(2002)
Hum Gene Ther
, vol.13
, pp. 163-175
-
-
Raper, S.E.1
Yudkoff, M.2
Chirmule, N.3
-
96
-
-
0142089747
-
Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer
-
Raper SE, Chirmule N, Lee FS, et al. 2003. Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer. Mol Genet Metab 80: 148-158.
-
(2003)
Mol Genet Metab
, vol.80
, pp. 148-158
-
-
Raper, S.E.1
Chirmule, N.2
Lee, F.S.3
-
97
-
-
40949120900
-
Full-length dystrophin gene transfer to the mdx mouse in utero
-
Reay DP, Bilbao R, Koppanati BM, et al. 2008. Full-length dystrophin gene transfer to the mdx mouse in utero. Gene Ther 15: 531-536.
-
(2008)
Gene Ther
, vol.15
, pp. 531-536
-
-
Reay, D.P.1
Bilbao, R.2
Koppanati, B.M.3
-
98
-
-
0034180256
-
Prenatal gene transfer; scientific, medical, and ethical issues
-
Recombinant DNA Advisory Committee.
-
Recombinant DNA Advisory Committee. 2000. Prenatal gene transfer; scientific, medical, and ethical issues. Hum Gene Ther 11: 1211-1229.
-
(2000)
Hum Gene Ther
, vol.11
, pp. 1211-1229
-
-
-
99
-
-
79959351886
-
A novel murine model of Cooley anemia and its rescue by lentiviral-mediated human â-globin gene transfer
-
Rivella S, May C, Chadburn A, Riviere I, Sadelain M. 2003. A novel murine model of Cooley anemia and its rescue by lentiviral-mediated human â-globin gene transfer. Science 294: 2368-2371.
-
(2003)
Science
, vol.294
, pp. 2368-2371
-
-
Rivella, S.1
May, C.2
Chadburn, A.3
Riviere, I.4
Sadelain, M.5
-
100
-
-
6344263893
-
Phase I study of dystrophin plasmid-based gene therapy in Duchenne/Becker muscular dystrophy
-
Romero NB, Benveniste O, Leturq F, et al. 2004. Phase I study of dystrophin plasmid-based gene therapy in Duchenne/Becker muscular dystrophy. Hum Gene Ther 15(11): 1065-1076.
-
(2004)
Hum Gene Ther
, vol.15
, Issue.11
, pp. 1065-1076
-
-
Romero, N.B.1
Benveniste, O.2
Leturq, F.3
-
101
-
-
3242886999
-
Rescue of enzyme deficiency in embryonic diaphragm in a mouse model of metabolic myopathy: Pompe disease
-
Rucker M, Fraites TJ, Porvasnik SL, et al. 2004. Rescue of enzyme deficiency in embryonic diaphragm in a mouse model of metabolic myopathy: Pompe disease. Development 131: 3007-3019.
-
(2004)
Development
, vol.131
, pp. 3007-3019
-
-
Rucker, M.1
Fraites, T.J.2
Porvasnik, S.L.3
-
102
-
-
77952575684
-
Combining keratinocyte growth factor transfection into the airways and tracheal occlusion in a fetal sheep model of congenital diaphragmatic hernia
-
Saada J, Oudrhiri N, Bonnard A, et al. 2010. Combining keratinocyte growth factor transfection into the airways and tracheal occlusion in a fetal sheep model of congenital diaphragmatic hernia. J Gene Med 12: 413-422.
-
(2010)
J Gene Med
, vol.12
, pp. 413-422
-
-
Saada, J.1
Oudrhiri, N.2
Bonnard, A.3
-
103
-
-
34548068776
-
Persistent expression of hFIX after tolerance induction by in utero or neonatal administration of AAV-1-F.IX in hemophilia B mice
-
Sabatino DE, MacKenzie TC, Peranteau WH, et al. 2007. Persistent expression of hFIX after tolerance induction by in utero or neonatal administration of AAV-1-F.IX in hemophilia B mice. Mol Ther 15: 1677-1685.
-
(2007)
Mol Ther
, vol.15
, pp. 1677-1685
-
-
Sabatino, D.E.1
MacKenzie, T.C.2
Peranteau, W.H.3
-
104
-
-
4744364706
-
Nonviral gene transfer to surface skin of mid-gestational murine embryos by intraamniotic injection and subsequent electroporation
-
Sato M, Tanigawa M, Kikuchi N. 2004. Nonviral gene transfer to surface skin of mid-gestational murine embryos by intraamniotic injection and subsequent electroporation. Mol Reprod Dev 69(268): 277.
-
(2004)
Mol Reprod Dev
, vol.69
, Issue.268
, pp. 277
-
-
Sato, M.1
Tanigawa, M.2
Kikuchi, N.3
-
105
-
-
4644245706
-
In utero transplantation of autologous and allogeneic fetal liver stem cells in ovine fetuses
-
Schoeberlein A, Holzgreve W, Dudler L, Hahn S, Surbek DV. 2004. In utero transplantation of autologous and allogeneic fetal liver stem cells in ovine fetuses. Am J Obstet Gynecol 191: 1030-1036.
-
(2004)
Am J Obstet Gynecol
, vol.191
, pp. 1030-1036
-
-
Schoeberlein, A.1
Holzgreve, W.2
Dudler, L.3
Hahn, S.4
Surbek, D.V.5
-
106
-
-
0034030812
-
Adenovirus-mediated in utero gene transfer in mice and guinea pigs: tissue distribution of recombinant adenovirus determined by quantitative TaqMan-polymerase chain reaction assay
-
Senoo M, Matsubara Y, Fujii K, et al. 2000. Adenovirus-mediated in utero gene transfer in mice and guinea pigs: tissue distribution of recombinant adenovirus determined by quantitative TaqMan-polymerase chain reaction assay. Mol Genet Metab 69: 269-276.
-
(2000)
Mol Genet Metab
, vol.69
, pp. 269-276
-
-
Senoo, M.1
Matsubara, Y.2
Fujii, K.3
-
107
-
-
33645779725
-
Immune response to lentiviral bilirubin UDP-glucuronosyl-transferase gene transfer in fetal and neonatal rats
-
Seppen J. 2006. Immune response to lentiviral bilirubin UDP-glucuronosyl-transferase gene transfer in fetal and neonatal rats. Gene Ther 13: 672-677.
-
(2006)
Gene Ther
, vol.13
, pp. 672-677
-
-
Seppen, J.1
-
108
-
-
0142181993
-
Long-term correction of bilirubin UDPglucuronyltransferase deficiency in rats by in utero lentiviral gene transfer
-
Seppen J, van der Rijt R, Looije N, van Til NP, Lamers WH, Elferink RPJO. 2003. Long-term correction of bilirubin UDPglucuronyltransferase deficiency in rats by in utero lentiviral gene transfer. Mol Ther 8: 593-599.
-
(2003)
Mol Ther
, vol.8
, pp. 593-599
-
-
Seppen, J.1
van der Rijt, R.2
Looije, N.3
van Til, N.P.4
Lamers, W.H.5
Elferink, R.P.J.O.6
-
109
-
-
79953146322
-
Autologous transplantation of amniotic fluid derived mesenchymal stem cells into sheep fetuses
-
in press).
-
Shaw SWS, Bollini S, Abi-Nader K, et al. 2010. Autologous transplantation of amniotic fluid derived mesenchymal stem cells into sheep fetuses. Cell Transplant (in press).
-
(2010)
Cell Transplant
-
-
Shaw, S.W.S.1
Bollini, S.2
Abi-Nader, K.3
-
110
-
-
79953155954
-
Clinical applications of prenatal and postnatal therapy using stem cells retrieved from amniotic fluid
-
Shaw SWS, David AL, De Coppi P. 2011. Clinical applications of prenatal and postnatal therapy using stem cells retrieved from amniotic fluid. Curr Opin Obstet Gynecol 23: 109-116.
-
(2011)
Curr Opin Obstet Gynecol
, vol.23
, pp. 109-116
-
-
Shaw, S.W.S.1
David, A.L.2
De Coppi, P.3
-
111
-
-
0036080633
-
Adenovirus-mediated prenatal gene transfer to murine central nervous system
-
Shen JS, Meng XL, Ohashi T, Eto Y. 2002. Adenovirus-mediated prenatal gene transfer to murine central nervous system. Gene Ther 9: 819-823.
-
(2002)
Gene Ther
, vol.9
, pp. 819-823
-
-
Shen, J.S.1
Meng, X.L.2
Ohashi, T.3
Eto, Y.4
-
112
-
-
13644258459
-
Widespread gene transduction to the central nervous system by adenovirus in utero. implication for prenatal gene therapy to brain involvement of lysosomal storage disease
-
Shen J, Meng X, Maeda H, Ohashi T, Eto Y. 2004. Widespread gene transduction to the central nervous system by adenovirus in utero. implication for prenatal gene therapy to brain involvement of lysosomal storage disease. J Gene Med 6: 1206-1215.
-
(2004)
J Gene Med
, vol.6
, pp. 1206-1215
-
-
Shen, J.1
Meng, X.2
Maeda, H.3
Ohashi, T.4
Eto, Y.5
-
113
-
-
0025487393
-
Evaluation of the transfer and expression in mice of an enzyme encoding gene using a human adenovirus vector
-
Stratford-Perricaudet L, Levrero M, Chasse J-F, Perricaudet M, Briand P. 1990. Evaluation of the transfer and expression in mice of an enzyme encoding gene using a human adenovirus vector. Hum Gene Ther 1: 241-256.
-
(1990)
Hum Gene Ther
, vol.1
, pp. 241-256
-
-
Stratford-Perricaudet, L.1
Levrero, M.2
Chasse, J.-F.3
Perricaudet, M.4
Briand, P.5
-
114
-
-
0030748567
-
Fetoscopic gene therapy for congenital lung disease
-
Sylvester KG, Yang EY, Cass DL, Crombleholme TM, Scott Adzick N. 1997. Fetoscopic gene therapy for congenital lung disease. J Pediatr Surg 7: 964-969.
-
(1997)
J Pediatr Surg
, vol.7
, pp. 964-969
-
-
Sylvester, K.G.1
Yang, E.Y.2
Cass, D.L.3
Crombleholme, T.M.4
Scott Adzick, N.5
-
116
-
-
33845788246
-
Fetal gene transfer using lentiviral vectors: in vivo detection of gene expression by microPET and optical imaging in fetal and infant monkeys
-
Tarantal AF. 2006. Fetal gene transfer using lentiviral vectors: in vivo detection of gene expression by microPET and optical imaging in fetal and infant monkeys. Hum Gene Ther 17: 1254-1261.
-
(2006)
Hum Gene Ther
, vol.17
, pp. 1254-1261
-
-
Tarantal, A.F.1
-
117
-
-
0027499715
-
Evaluation of the bioeffects of prenatal ultrasound exposure in the cynomolgus macaque (Macaca fascicularis): III. Developmental and hematologic studies
-
Tarantal AF, O'Brien WD, Hendrickx AG. 1993. Evaluation of the bioeffects of prenatal ultrasound exposure in the cynomolgus macaque (Macaca fascicularis): III. Developmental and hematologic studies. Teratology 47: 159-170.
-
(1993)
Teratology
, vol.47
, pp. 159-170
-
-
Tarantal, A.F.1
O'Brien, W.D.2
Hendrickx, A.G.3
-
118
-
-
0035668699
-
Lentiviral vector gene transfer into fetal rhesus monkeys (Macaca mulatta): lung-targeting approaches
-
Tarantal AF, Lee CI, Ekert JE, et al. 2001a. Lentiviral vector gene transfer into fetal rhesus monkeys (Macaca mulatta): lung-targeting approaches. Mol Ther 4: 614-621.
-
(2001)
Mol Ther
, vol.4
, pp. 614-621
-
-
Tarantal, A.F.1
Lee, C.I.2
Ekert, J.E.3
-
119
-
-
79959370990
-
Lentiviral vector gene transfer efficiency using fetal routes of administration in Rhesus monkeys
-
Tarantal AF, Lee CI, Ekert JE, et al. 2001b. Lentiviral vector gene transfer efficiency using fetal routes of administration in Rhesus monkeys. Mol Ther 3: S129.
-
(2001)
Mol Ther
, vol.3
-
-
Tarantal, A.F.1
Lee, C.I.2
Ekert, J.E.3
-
120
-
-
0034985713
-
Rhesus monkey model for fetal gene transfer: studies with retroviral-based vector systems
-
Tarantal AF, O'Rourke JP, Case SS, et al. 2001c. Rhesus monkey model for fetal gene transfer: studies with retroviral-based vector systems. Mol Ther 3: 128-138.
-
(2001)
Mol Ther
, vol.3
, pp. 128-138
-
-
Tarantal, A.F.1
O'Rourke, J.P.2
Case, S.S.3
-
121
-
-
20844455351
-
Intrapulmonary and intramyocardial gene transfer in rhesus monkeys (Macaca mulatta): safety and efficiency of HIV-1-derived lentiviral vectors for fetal gene delivery
-
Tarantal AF, McDonald RJ, Jimenez DF, et al. 2005. Intrapulmonary and intramyocardial gene transfer in rhesus monkeys (Macaca mulatta): safety and efficiency of HIV-1-derived lentiviral vectors for fetal gene delivery. Mol Ther 12: 87-98.
-
(2005)
Mol Ther
, vol.12
, pp. 87-98
-
-
Tarantal, A.F.1
McDonald, R.J.2
Jimenez, D.F.3
-
122
-
-
77957840310
-
Overexpression of transforming growth factor-beta1 in fetal monkey lung results in prenatal pulmonary fibrosis
-
Tarantal AF, Chen H, Shi TT, et al. 2010. Overexpression of transforming growth factor-beta1 in fetal monkey lung results in prenatal pulmonary fibrosis. Eur Respir J 36(4): 907-914.
-
(2010)
Eur Respir J
, vol.36
, Issue.4
, pp. 907-914
-
-
Tarantal, A.F.1
Chen, H.2
Shi, T.T.3
-
123
-
-
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. 1999. 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 6: 1239-1248.
-
(1999)
Gene Ther
, vol.6
, pp. 1239-1248
-
-
Themis, M.1
Schneider, H.2
Kiserud, T.3
-
124
-
-
79959354925
-
Long term pulmonary gene therapy with a lentiviral vector in a fetal rat model
-
Toelen J, Rik G, Sbragia L, Christophe D, Debyser Z, Deprest J. 2006. Long term pulmonary gene therapy with a lentiviral vector in a fetal rat model. Am J Obstet Gynecol 95: S22.
-
(2006)
Am J Obstet Gynecol
, vol.95
-
-
Toelen, J.1
Rik, G.2
Sbragia, L.3
Christophe, D.4
Debyser, Z.5
Deprest, J.6
-
125
-
-
35348897210
-
Immunological lessons learnt from patients transplanted with fully mismatched stem cells
-
Touraine J-L, Plotnicky H, Roncarolo MG, Bachetta R, Gebuhrer L. 2007. Immunological lessons learnt from patients transplanted with fully mismatched stem cells. Immunol Res 38: 201-209.
-
(2007)
Immunol Res
, vol.38
, pp. 201-209
-
-
Touraine, J.-L.1
Plotnicky, H.2
Roncarolo, M.G.3
Bachetta, R.4
Gebuhrer, L.5
-
126
-
-
0035383766
-
Induction of stable prenatal tolerance to β-galactosidase by in utero gene transfer into preimmune sheep fetuses
-
Tran ND, Porada CD, Almeida-Porada G, Glimp HA, Anderson WF, Zanjani ED. 2001. Induction of stable prenatal tolerance to β-galactosidase by in utero gene transfer into preimmune sheep fetuses. Blood 97: 3417-3423.
-
(2001)
Blood
, vol.97
, pp. 3417-3423
-
-
Tran, N.D.1
Porada, C.D.2
Almeida-Porada, G.3
Glimp, H.A.4
Anderson, W.F.5
Zanjani, E.D.6
-
127
-
-
5444227748
-
Permanent phenotypic correction of hemophilia B in immunocompetent mice by prenatal gene therapy
-
Waddington SN, Nivsarkar MS, Mistry AR, et al. 2004. Permanent phenotypic correction of hemophilia B in immunocompetent mice by prenatal gene therapy. Blood 104: 2714-2721.
-
(2004)
Blood
, vol.104
, pp. 2714-2721
-
-
Waddington, S.N.1
Nivsarkar, M.S.2
Mistry, A.R.3
-
128
-
-
35348922932
-
Fetal gene transfer
-
Waddington SN, Buckley SM, David AL, Peebles DM, Rodeck CH, Coutelle C. 2007. Fetal gene transfer. Curr Opin Mol Ther 9: 432-438.
-
(2007)
Curr Opin Mol Ther
, vol.9
, pp. 432-438
-
-
Waddington, S.N.1
Buckley, S.M.2
David, A.L.3
Peebles, D.M.4
Rodeck, C.H.5
Coutelle, C.6
-
129
-
-
0034610364
-
Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model
-
Wang B, Li J, Xiao X. 2000. Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model. Proc Natl Acad Sci Am 97: 13714-13719.
-
(2000)
Proc Natl Acad Sci Am
, vol.97
, pp. 13714-13719
-
-
Wang, B.1
Li, J.2
Xiao, X.3
-
130
-
-
0034986080
-
Use of perfluorochemical liquid allows earlier detection of gene expression and use of less vector in normal lung and enhances gene expression in acutely injured lung
-
Weiss DJ, Bonneau L, Liggitt D. 2001. Use of perfluorochemical liquid allows earlier detection of gene expression and use of less vector in normal lung and enhances gene expression in acutely injured lung. Mol Ther 3: 734-745.
-
(2001)
Mol Ther
, vol.3
, pp. 734-745
-
-
Weiss, D.J.1
Bonneau, L.2
Liggitt, D.3
-
131
-
-
24644444255
-
Targeting the respiratory muscles of fetal sheep for prenatal gene therapy for Duchenne muscular dystrophy
-
Weisz B, David AL, Gregory LG, et al. 2005. Targeting the respiratory muscles of fetal sheep for prenatal gene therapy for Duchenne muscular dystrophy. Am J Obstet Gynecol 193: 1105-1109.
-
(2005)
Am J Obstet Gynecol
, vol.193
, pp. 1105-1109
-
-
Weisz, B.1
David, A.L.2
Gregory, L.G.3
-
132
-
-
85012550859
-
Immunological hurdles in the path to gene therapy for Duchenne muscular dystrophy
-
Wells DJ, Ferrrer A, Wells KE. 2002. Immunological hurdles in the path to gene therapy for Duchenne muscular dystrophy. Expert Rev Mol Med 4(23): 1-23.
-
(2002)
Expert Rev Mol Med
, vol.4
, Issue.23
, pp. 1-23
-
-
Wells, D.J.1
Ferrrer, A.2
Wells, K.E.3
-
134
-
-
26944481086
-
CpG-island fragments from the HNRPA2B1/CBX3 genomic locus reduce silencing and enhance transgene expression from the hCMV promoter/enhancer in mammalian cells
-
Williams S, Mustoe T, Mulcahy T, et al. 2005. CpG-island fragments from the HNRPA2B1/CBX3 genomic locus reduce silencing and enhance transgene expression from the hCMV promoter/enhancer in mammalian cells. BMC Biotechnol 5: 17.
-
(2005)
BMC Biotechnol
, vol.5
, pp. 17
-
-
Williams, S.1
Mustoe, T.2
Mulcahy, T.3
-
135
-
-
33745608443
-
Lentiviral expression of retinal guanylate cyclase-1 (RetGC1) restores vision in an avian model of childhood blindness
-
Williams ML, Coleman JE, Haire SE, et al. 2006. Lentiviral expression of retinal guanylate cyclase-1 (RetGC1) restores vision in an avian model of childhood blindness. PLoS Med 3: e201.
-
(2006)
PLoS Med
, vol.3
-
-
Williams, M.L.1
Coleman, J.E.2
Haire, S.E.3
-
136
-
-
0033428380
-
Prenatal orogastric gene delivery results in transduction of the small bowel in the fetal rabbit
-
Wu Y, Liu J, Woo S, Finegold MJ, Brandt ML. 1999. Prenatal orogastric gene delivery results in transduction of the small bowel in the fetal rabbit. Fetal Diagn Ther 14: 323-327.
-
(1999)
Fetal Diagn Ther
, vol.14
, pp. 323-327
-
-
Wu, Y.1
Liu, J.2
Woo, S.3
Finegold, M.J.4
Brandt, M.L.5
-
137
-
-
0033072104
-
Fetal gene therapy: efficacy, toxicity, and immunologic effects of early gestation recombinant adenovirus
-
Yang EY, Cass DL, Sylvester KG, Wilson JM, Adzick NS. 1999. Fetal gene therapy: efficacy, toxicity, and immunologic effects of early gestation recombinant adenovirus. J Pediatr Surg 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
-
138
-
-
9144231237
-
Successful in utero gene transfer using a gene gun in midgestational mouse fetuses
-
Yoshizawa J, Li X-K, Fujino M, et al. 2004. Successful in utero gene transfer using a gene gun in midgestational mouse fetuses. J Pediatr Surg 39: 81-84.
-
(2004)
J Pediatr Surg
, vol.39
, pp. 81-84
-
-
Yoshizawa, J.1
Li, X.-K.2
Fujino, M.3
-
139
-
-
34548843603
-
Lentiviral vectors containing an enhancer-less ubiquitously acting chromatin opening element (UCOE) provide highly reproducible and stable transgene expression in hematopoietic cells
-
Zhang F, Thornhill SI, Howe SJ, et al. 2007. Lentiviral vectors containing an enhancer-less ubiquitously acting chromatin opening element (UCOE) provide highly reproducible and stable transgene expression in hematopoietic cells. Blood 110: 1448-1457.
-
(2007)
Blood
, vol.110
, pp. 1448-1457
-
-
Zhang, F.1
Thornhill, S.I.2
Howe, S.J.3
|