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Volumn 22, Issue 1, 2008, Pages 203-218

Gene therapy for the fetus: is there a future?

Author keywords

congenital disease; fetus; gene therapy; gene transfer

Indexed keywords

ADENOVIRUS VECTOR; BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 9; LENTIVIRUS VECTOR; LIPOSOME; NAKED DNA; PARVOVIRUS VECTOR; POLYETHYLENEIMINE; RETROVIRUS VECTOR; VIRUS VECTOR;

EID: 39049091406     PISSN: 15216934     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.bpobgyn.2007.08.008     Document Type: Review
Times cited : (55)

References (88)
  • 1
    • 0028807727 scopus 로고
    • T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years
    • Blaese R.M., Culver K.W., Miller A.D., et al. T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years. Science 270 (1995) 475-480
    • (1995) Science , vol.270 , pp. 475-480
    • Blaese, R.M.1    Culver, K.W.2    Miller, A.D.3
  • 3
    • 0347003513 scopus 로고    scopus 로고
    • The burden of genetic disease on inpatient care in a children's hospital
    • McCandless S.E., Brunger J.W., and Cassidy S.B. The burden of genetic disease on inpatient care in a children's hospital. Am J Hum Genet 74 (2004) 121-127
    • (2004) Am J Hum Genet , vol.74 , pp. 121-127
    • McCandless, S.E.1    Brunger, J.W.2    Cassidy, S.B.3
  • 4
    • 0029028833 scopus 로고
    • The challenge of fetal gene therapy
    • Coutelle C., Douar A.-M., Colledge W.H., et al. The challenge of fetal gene therapy. Nat Med 1 (1995) 864-866
    • (1995) Nat Med , vol.1 , pp. 864-866
    • Coutelle, C.1    Douar, A.-M.2    Colledge, W.H.3
  • 5
    • 0042674017 scopus 로고    scopus 로고
    • Long-term transgene expression by administration of a lentivirus-based vector to the fetal circulation of immuno-competent mice
    • Waddington S.N., Mitrophanous K.A., Ellard F., et al. Long-term transgene expression by administration of a lentivirus-based vector to the fetal circulation of immuno-competent mice. Gene Ther 10 (2003) 1234-1240
    • (2003) Gene Ther , vol.10 , pp. 1234-1240
    • Waddington, S.N.1    Mitrophanous, K.A.2    Ellard, F.3
  • 6
    • 0346035738 scopus 로고    scopus 로고
    • Efficient transduction of liver and muscle after in utero injection of lentiviral vectors with different pseudotypes
    • MacKenzie T.C., Kobinger G.P., Kootstra N.A., et al. Efficient transduction of liver and muscle after in utero injection of lentiviral vectors with different pseudotypes. Mol Ther 6 (2002) 349-358
    • (2002) Mol Ther , vol.6 , pp. 349-358
    • MacKenzie, T.C.1    Kobinger, G.P.2    Kootstra, N.A.3
  • 7
    • 0037372398 scopus 로고    scopus 로고
    • Therapeutic factor VIII levels and negligible toxicity in mouse and dog models of hemophilia A following gene therapy with high-capacity adenoviral vectors
    • Chuah M.K., 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 101 (2003) 1734-1743
    • (2003) Blood , vol.101 , pp. 1734-1743
    • Chuah, M.K.1    Schiedner, G.2    Thorrez, L.3
  • 8
    • 5444275097 scopus 로고    scopus 로고
    • Immune responses to gene therapy vectors: influence on vector function and effector mechanisms
    • Bessis N., GarciaCozar F.J., and Boissier M.C. Immune responses to gene therapy vectors: influence on vector function and effector mechanisms. Gene Ther 11 (2004) S10-S17
    • (2004) Gene Ther , vol.11
    • Bessis, N.1    GarciaCozar, F.J.2    Boissier, M.C.3
  • 9
    • 0347927259 scopus 로고    scopus 로고
    • Immune response to full-length dystrophin delivered to Dmd muscle by a high-capacity adenoviral vector
    • Gilchrist S.C., Ontell M.P., Kochanek S., et al. Immune response to full-length dystrophin delivered to Dmd muscle by a high-capacity adenoviral vector. Mol Ther 6 (2002) 359-368
    • (2002) Mol Ther , vol.6 , pp. 359-368
    • Gilchrist, S.C.1    Ontell, M.P.2    Kochanek, S.3
  • 10
    • 0037344452 scopus 로고    scopus 로고
    • Long-term normalization in the central nervous system, ocular manifestations, and skeletal deformities by a single systemic adenovirus injection into neonatal mice with mucopolysaccharidosis VII
    • Kamata Y., Tanabe A., Kanaji A., et al. Long-term normalization in the central nervous system, ocular manifestations, and skeletal deformities by a single systemic adenovirus injection into neonatal mice with mucopolysaccharidosis VII. Gene Ther 10 (2003) 406-414
    • (2003) Gene Ther , vol.10 , pp. 406-414
    • Kamata, Y.1    Tanabe, A.2    Kanaji, A.3
  • 11
    • 0042526080 scopus 로고    scopus 로고
    • Gene therapy progress and prospects: gene therapy of lysosomal storage disorders
    • Cheng S.H., and Smith A.E. Gene therapy progress and prospects: gene therapy of lysosomal storage disorders. Gene Ther 10 (2003) 1275-1281
    • (2003) Gene Ther , vol.10 , pp. 1275-1281
    • Cheng, S.H.1    Smith, A.E.2
  • 12
    • 5444227748 scopus 로고    scopus 로고
    • Permanent phenotypic correction of Hemophilia B in immunocompetent mice by prenatal gene therapy
    • Waddington S.N., Nivsarkar M.S., Mistry A.R., et al. Permanent phenotypic correction of Hemophilia B in immunocompetent mice by prenatal gene therapy. Blood 104 (2004) 2714-2721
    • (2004) Blood , vol.104 , pp. 2714-2721
    • Waddington, S.N.1    Nivsarkar, M.S.2    Mistry, A.R.3
  • 13
    • 0033093921 scopus 로고    scopus 로고
    • Report on the potential use of gene therapy in utero. Gene Therapy Advisory Committee
    • Wilson J.M., and Wivel N.A. Report on the potential use of gene therapy in utero. Gene Therapy Advisory Committee. Hum Gene Ther 10 (1999) 689-692
    • (1999) Hum Gene Ther , vol.10 , pp. 689-692
    • Wilson, J.M.1    Wivel, N.A.2
  • 14
    • 0033539486 scopus 로고    scopus 로고
    • Short-term correction of factor VIII deficiency in a murine model of hemophilia A after delivery of adenovirus murine factor VIII in utero
    • Lipshutz G.S., Sarkar R., Flebbe-Rehwaldt L., 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 U S A 96 (1999) 13324-13329
    • (1999) Proc Natl Acad Sci U S A , vol.96 , pp. 13324-13329
    • Lipshutz, G.S.1    Sarkar, R.2    Flebbe-Rehwaldt, L.3
  • 15
    • 10744230959 scopus 로고    scopus 로고
    • In utero gene therapy rescues vision in a murine model of congenital blindness
    • Dejneka N.S., Surace E.M., Aleman T.S., et al. In utero gene therapy rescues vision in a murine model of congenital blindness. Mol Ther 9 (2004) 182-188
    • (2004) Mol Ther , vol.9 , pp. 182-188
    • Dejneka, N.S.1    Surace, E.M.2    Aleman, T.S.3
  • 16
    • 0142181993 scopus 로고    scopus 로고
    • Long-term correction of bilirubin UDPglucuronyltransferase deficiency in rats by in utero lentiviral gene transfer
    • Seppen J., van der Rijt R., Looije N., et al. Long-term correction of bilirubin UDPglucuronyltransferase deficiency in rats by in utero lentiviral gene transfer. Mol Ther 8 (2003) 593-599
    • (2003) Mol Ther , vol.8 , pp. 593-599
    • Seppen, J.1    van der Rijt, R.2    Looije, N.3
  • 17
    • 3242886999 scopus 로고    scopus 로고
    • Rescue of enzyme deficiency in embryonic diaphragm in a mouse model of metabolic myopathy: Pompe disease
    • Rucker M., Fraites T.J., Porvasnik S.L., et al. Rescue of enzyme deficiency in embryonic diaphragm in a mouse model of metabolic myopathy: Pompe disease. Development 131 (2004) 3007-3019
    • (2004) Development , vol.131 , pp. 3007-3019
    • Rucker, M.1    Fraites, T.J.2    Porvasnik, S.L.3
  • 18
    • 0028334619 scopus 로고
    • A practical guide to the evaluation and treatment of hemophilia
    • Furie B., Limentani S.A., and Rosenfield C.G. A practical guide to the evaluation and treatment of hemophilia. Blood 84 (1994) 3-9
    • (1994) Blood , vol.84 , pp. 3-9
    • Furie, B.1    Limentani, S.A.2    Rosenfield, C.G.3
  • 20
    • 0034661520 scopus 로고    scopus 로고
    • Mortality among males with hemophilia: relations with source of medical care
    • The Hemophilia Surveillance System Project Investigators
    • Soucie J.M., Nuss R., Evatt B., et al. Mortality among males with hemophilia: relations with source of medical care. The Hemophilia Surveillance System Project Investigators. Blood 96 (2000) 437-442
    • (2000) Blood , vol.96 , pp. 437-442
    • Soucie, J.M.1    Nuss, R.2    Evatt, B.3
  • 21
    • 17444372024 scopus 로고    scopus 로고
    • Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors
    • Snyder R.O., Miao C., Meuse L., et al. Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors. Nat Med 5 (1999) 64-70
    • (1999) Nat Med , vol.5 , pp. 64-70
    • Snyder, R.O.1    Miao, C.2    Meuse, L.3
  • 22
    • 33644820684 scopus 로고    scopus 로고
    • Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response
    • Manno C.S., Pierce G.F., Arruda V.R., et al. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med 12 (2006) 342-347
    • (2006) Nat Med , vol.12 , pp. 342-347
    • Manno, C.S.1    Pierce, G.F.2    Arruda, V.R.3
  • 23
    • 0013442744 scopus 로고    scopus 로고
    • AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B
    • Manno C.S., Chew A.J., Hutchison S., et al. AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood 101 (2003) 2963-2972
    • (2003) Blood , vol.101 , pp. 2963-2972
    • Manno, C.S.1    Chew, A.J.2    Hutchison, S.3
  • 24
    • 0038442898 scopus 로고    scopus 로고
    • Nonviral gene transfer into fetal mouse livers (a comparison between the cationic polymer PEI and naked DNA)
    • Gharwan H., Wightman L., Kircheis R., et al. Nonviral gene transfer into fetal mouse livers (a comparison between the cationic polymer PEI and naked DNA). Gene Ther 10 (2003) 810-817
    • (2003) Gene Ther , vol.10 , pp. 810-817
    • Gharwan, H.1    Wightman, L.2    Kircheis, R.3
  • 25
    • 7444271942 scopus 로고    scopus 로고
    • Gene transfection into fetal sheep airways in utero using guanidinium-cholesterol cationic lipids
    • Luton D., Oudrhiri N., De Lagausie P., et al. Gene transfection into fetal sheep airways in utero using guanidinium-cholesterol cationic lipids. J Gene Med 6 (2004) 328-336
    • (2004) J Gene Med , vol.6 , pp. 328-336
    • Luton, D.1    Oudrhiri, N.2    De Lagausie, P.3
  • 26
    • 30444443146 scopus 로고    scopus 로고
    • In vivo studies of dialkynoyl analogues of DOTAP demonstrate improved gene transfer efficiency of cationic liposomes in mouse lung
    • Fletcher S., Ahmad A., Perouzel E., et al. In vivo studies of dialkynoyl analogues of DOTAP demonstrate improved gene transfer efficiency of cationic liposomes in mouse lung. J Med Chem 49 (2006) 349-357
    • (2006) J Med Chem , vol.49 , pp. 349-357
    • Fletcher, S.1    Ahmad, A.2    Perouzel, E.3
  • 27
    • 0032925992 scopus 로고    scopus 로고
    • Gene transfer in utero biologically engineers a patent ductus arteriosus in lambs by arresting fibronectin-dependent neointimal formation
    • Mason C.A., Bigras J.L., O'Blenes S.B., et al. Gene transfer in utero biologically engineers a patent ductus arteriosus in lambs by arresting fibronectin-dependent neointimal formation. Nat Med 5 (1999) 176-182
    • (1999) Nat Med , vol.5 , pp. 176-182
    • Mason, C.A.1    Bigras, J.L.2    O'Blenes, S.B.3
  • 28
    • 0037338159 scopus 로고    scopus 로고
    • Ultrasound guided percutaneous delivery of adenoviral vectors encoding the b-galactosidase and human factor IX genes to early gestation fetal sheep in utero
    • David A.L., Cook T., Waddington S., et al. Ultrasound guided percutaneous delivery of adenoviral vectors encoding the b-galactosidase and human factor IX genes to early gestation fetal sheep in utero. Hum Gene Ther 14 (2003) 353-364
    • (2003) Hum Gene Ther , vol.14 , pp. 353-364
    • David, A.L.1    Cook, T.2    Waddington, S.3
  • 29
    • 0037441596 scopus 로고    scopus 로고
    • In utero gene transfer of human factor IX to fetal mice can induce postnatal tolerance of the exogenous clotting factor
    • Waddington S.N., Buckley S.M.K., Nivsarkar M., et al. In utero gene transfer of human factor IX to fetal mice can induce postnatal tolerance of the exogenous clotting factor. Blood 101 (2003) 1359-1366
    • (2003) Blood , vol.101 , pp. 1359-1366
    • Waddington, S.N.1    Buckley, S.M.K.2    Nivsarkar, M.3
  • 30
    • 0034294530 scopus 로고    scopus 로고
    • Reexpression following readministration of an adenoviral vector in adult mice after initial in utero adenoviral administration
    • Lipshutz G.S., Flebbe-Rehwaldt L., and Gaensler K.M.L. Reexpression following readministration of an adenoviral vector in adult mice after initial in utero adenoviral administration. Mol Ther 2 (2000) 374-380
    • (2000) Mol Ther , vol.2 , pp. 374-380
    • Lipshutz, G.S.1    Flebbe-Rehwaldt, L.2    Gaensler, K.M.L.3
  • 31
    • 0029046114 scopus 로고
    • Adenoviral-mediated gene transfer to fetal pulmonary epithelia in vitro and in vivo
    • McCray P.B., Armstrong K., Zabner J., et al. Adenoviral-mediated gene transfer to fetal pulmonary epithelia in vitro and in vivo. J Clin Invest 95 (1995) 2620-2632
    • (1995) J Clin Invest , vol.95 , pp. 2620-2632
    • McCray, P.B.1    Armstrong, K.2    Zabner, J.3
  • 32
    • 0031049699 scopus 로고    scopus 로고
    • Persistence in muscle of an adenoviral vector that lacks all viral genes
    • Chen H.H., Mack L.M., Kelly R., et al. Persistence in muscle of an adenoviral vector that lacks all viral genes. Proc Natl Acad Sci U S A 94 (1997) 1645-1650
    • (1997) Proc Natl Acad Sci U S A , vol.94 , pp. 1645-1650
    • Chen, H.H.1    Mack, L.M.2    Kelly, R.3
  • 33
    • 0031916435 scopus 로고    scopus 로고
    • Genomic DNA transfer with a high-capacity adenovirus vector results in improved in vivo gene expression and decreased toxicity
    • Schiedner G., Morral N., Parks R.J., et al. Genomic DNA transfer with a high-capacity adenovirus vector results in improved in vivo gene expression and decreased toxicity. Nat Genet 18 (1998) 180-183
    • (1998) Nat Genet , vol.18 , pp. 180-183
    • Schiedner, G.1    Morral, N.2    Parks, R.J.3
  • 34
    • 0036210482 scopus 로고    scopus 로고
    • Novel integrating adenoviral/retroviral hybrid vector for gene therapy
    • Murphy S.J., Chong H., Bell S., et al. Novel integrating adenoviral/retroviral hybrid vector for gene therapy. Hum Gene Ther 13 (2002) 745-760
    • (2002) Hum Gene Ther , vol.13 , pp. 745-760
    • Murphy, S.J.1    Chong, H.2    Bell, S.3
  • 35
    • 0037370612 scopus 로고    scopus 로고
    • A new hybrid system capable of efficient lentiviral vector production and stable gene transfer mediated by a single helper-dependent adenoviral vector
    • Kubo S., and Mitani K. A new hybrid system capable of efficient lentiviral vector production and stable gene transfer mediated by a single helper-dependent adenoviral vector. J Virol 77 (2003) 2964-2971
    • (2003) J Virol , vol.77 , pp. 2964-2971
    • Kubo, S.1    Mitani, K.2
  • 36
    • 0036081135 scopus 로고    scopus 로고
    • Humoral immune response to recombinant adenovirus and adeno-associated virus after in utero administration of viral vectors in mice
    • Jerebtsova M., Batshaw M.L., and Ye X. Humoral immune response to recombinant adenovirus and adeno-associated virus after in utero administration of viral vectors in mice. Pediatr Res 52 (2002) 95-104
    • (2002) Pediatr Res , vol.52 , pp. 95-104
    • Jerebtsova, M.1    Batshaw, M.L.2    Ye, X.3
  • 37
    • 24444479621 scopus 로고    scopus 로고
    • Prolonged expression of adenovirus and adeno-associated virus following fetal administration in the preimmune period
    • Bouchard S. Prolonged expression of adenovirus and adeno-associated virus following fetal administration in the preimmune period. Mol Ther 3 (2001) S393-S394
    • (2001) Mol Ther , vol.3
    • Bouchard, S.1
  • 38
    • 0036371236 scopus 로고    scopus 로고
    • Sustained delivery of therapeutic concentrations of human clotting factor IX - a comparison of adenoviral and AAV vectors administered in utero
    • Schneider H., Mühle C., Douar A.-M., et al. Sustained delivery of therapeutic concentrations of human clotting factor IX - a comparison of adenoviral and AAV vectors administered in utero. J Gene Med 4 (2002) 46-53
    • (2002) J Gene Med , vol.4 , pp. 46-53
    • Schneider, H.1    Mühle, C.2    Douar, A.-M.3
  • 39
    • 0041698415 scopus 로고    scopus 로고
    • Comparison of gene expression after intraperitoneal delivery of AAV2 or AAV5 in utero
    • Lipshutz G.S., Titre D., Brindle M., et al. Comparison of gene expression after intraperitoneal delivery of AAV2 or AAV5 in utero. Mol Ther 8 (2003) 90-98
    • (2003) Mol Ther , vol.8 , pp. 90-98
    • Lipshutz, G.S.1    Titre, D.2    Brindle, M.3
  • 40
    • 4243142741 scopus 로고    scopus 로고
    • Long term modification of adult physiology following in utero gene transfer using an AAV vector
    • Garrett D.J., Cohen C.J., and Larson J.E. Long term modification of adult physiology following in utero gene transfer using an AAV vector. Genet Vaccines Ther 2 (2004) 4
    • (2004) Genet Vaccines Ther , vol.2 , pp. 4
    • Garrett, D.J.1    Cohen, C.J.2    Larson, J.E.3
  • 41
    • 0038054338 scopus 로고    scopus 로고
    • AAV serotype 2 vectors preferentially integrate into active genes in mice
    • Nakai H., Montini E., Fuess S., et al. AAV serotype 2 vectors preferentially integrate into active genes in mice. Nat Genet 34 (2003) 297-302
    • (2003) Nat Genet , vol.34 , pp. 297-302
    • Nakai, H.1    Montini, E.2    Fuess, S.3
  • 43
    • 0032834295 scopus 로고    scopus 로고
    • Evidence for infection of the human embryo with adeno-associated virus in pregnancy
    • Burguete T., Rabreau M., Fontanges-Darriet M., et al. Evidence for infection of the human embryo with adeno-associated virus in pregnancy. Hum Reprod 14 (1999) 2396-2401
    • (1999) Hum Reprod , vol.14 , pp. 2396-2401
    • Burguete, T.1    Rabreau, M.2    Fontanges-Darriet, M.3
  • 44
    • 0026510716 scopus 로고
    • Widespread dispersion of neuronal clones across functional regions of the cerebral cortex
    • Walsh C., and Cepko C.L. Widespread dispersion of neuronal clones across functional regions of the cerebral cortex. Science 255 (1992) 434-441
    • (1992) Science , vol.255 , pp. 434-441
    • Walsh, C.1    Cepko, C.L.2
  • 45
    • 0025064567 scopus 로고
    • Hepatic gene transfer in animals using retroviruses containing the promoter from the gene for phosphoenolpyruvate carboxykinase
    • Hatzoglou M., Lamers W., Bosch F., et al. Hepatic gene transfer in animals using retroviruses containing the promoter from the gene for phosphoenolpyruvate carboxykinase. J Biol 265 (1990) 17285-17293
    • (1990) J Biol , vol.265 , pp. 17285-17293
    • Hatzoglou, M.1    Lamers, W.2    Bosch, F.3
  • 46
    • 0032551689 scopus 로고    scopus 로고
    • In utero gene therapy: transfer and long-term expression of the bacterial neor gene in sheep after direct injection of retroviral vectors into preimmune fetuses
    • Porada C.D., Tran N., Eglitis M., et al. In utero gene therapy: transfer and long-term expression of the bacterial neor gene in sheep after direct injection of retroviral vectors into preimmune fetuses. Hum Gene Ther 9 (1998) 1571-1585
    • (1998) Hum Gene Ther , vol.9 , pp. 1571-1585
    • Porada, C.D.1    Tran, N.2    Eglitis, M.3
  • 47
    • 0034985713 scopus 로고    scopus 로고
    • Rhesus monkey model for fetal gene transfer: studies with retroviral-based vector systems
    • Tarantal A.F., O'Rourke J.P., Case S.S., et al. Rhesus monkey model for fetal gene transfer: studies with retroviral-based vector systems. Mol Ther 3 (2001) 128-138
    • (2001) Mol Ther , vol.3 , pp. 128-138
    • Tarantal, A.F.1    O'Rourke, J.P.2    Case, S.S.3
  • 48
    • 0025279872 scopus 로고
    • Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection
    • Miller D.G., Adam M.A., and Miller A.D. Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection. Mol Cell Biol 10 (1990) 4239-4242
    • (1990) Mol Cell Biol , vol.10 , pp. 4239-4242
    • Miller, D.G.1    Adam, M.A.2    Miller, A.D.3
  • 49
    • 0016817004 scopus 로고
    • Human serum lyses RNA tumor viruses
    • Welsh R.J., Cooper N.R., Jensen F.C., et al. Human serum lyses RNA tumor viruses. Nature 257 (1975) 612-614
    • (1975) Nature , vol.257 , pp. 612-614
    • Welsh, R.J.1    Cooper, N.R.2    Jensen, F.C.3
  • 50
    • 0034882342 scopus 로고    scopus 로고
    • Targeted gene transfer to lymphocytes using murine leukaemia virus vectors pseudotyped with spleen necrosis virus envelope proteins
    • Engelstädter M., Buchholz C.J., Bobkova M., et al. Targeted gene transfer to lymphocytes using murine leukaemia virus vectors pseudotyped with spleen necrosis virus envelope proteins. Gene Ther 8 (2001) 1202-1206
    • (2001) Gene Ther , vol.8 , pp. 1202-1206
    • Engelstädter, M.1    Buchholz, C.J.2    Bobkova, M.3
  • 51
    • 0029793791 scopus 로고    scopus 로고
    • Effect of amniotic fluid on cationic lipid mediated transfection and viral infection
    • Douar A.-M., Themis M., Sandig V., et al. Effect of amniotic fluid on cationic lipid mediated transfection and viral infection. Gene Ther 3 (1996) 789-796
    • (1996) Gene Ther , vol.3 , pp. 789-796
    • Douar, A.-M.1    Themis, M.2    Sandig, V.3
  • 52
    • 0036677980 scopus 로고    scopus 로고
    • Inefficient transduction of sheep in utero after intra-amniotic injection of retroviral producer cells
    • Galan H.L., Bennett M.L., Tyson R.W., et al. Inefficient transduction of sheep in utero after intra-amniotic injection of retroviral producer cells. Am J Obstet Gynecol 187 (2002) 469-474
    • (2002) Am J Obstet Gynecol , vol.187 , pp. 469-474
    • Galan, H.L.1    Bennett, M.L.2    Tyson, R.W.3
  • 53
    • 0034784067 scopus 로고    scopus 로고
    • In utero gene delivery by intraamniotic injection of a retroviral vector producer cell line in a nonhuman primate model
    • Bennett M., Galan H., Owens G., et al. In utero gene delivery by intraamniotic injection of a retroviral vector producer cell line in a nonhuman primate model. Hum Gene Ther 12 (2001) 1857-1865
    • (2001) Hum Gene Ther , vol.12 , pp. 1857-1865
    • Bennett, M.1    Galan, H.2    Owens, G.3
  • 54
    • 0026654682 scopus 로고
    • Human immunodeficiency virus infection of cells arrested in the cell cycle
    • Lewis P., Hensel M., and Emerman M. Human immunodeficiency virus infection of cells arrested in the cell cycle. EMBO J 11 (1992) 3053-3058
    • (1992) EMBO J , vol.11 , pp. 3053-3058
    • Lewis, P.1    Hensel, M.2    Emerman, M.3
  • 55
    • 0033986340 scopus 로고    scopus 로고
    • Efficient lentiviral transduction of liver requires cell cycling in vivo
    • Park F., Ohashi K., Chiu W., et al. Efficient lentiviral transduction of liver requires cell cycling in vivo. Nat Genet 24 (2000) 49-52
    • (2000) Nat Genet , vol.24 , pp. 49-52
    • Park, F.1    Ohashi, K.2    Chiu, W.3
  • 56
    • 33746558043 scopus 로고    scopus 로고
    • Ultrasound-guided injection and occlusion of the trachea in fetal sheep
    • David A.L., Weisz B., Gregory L., et al. Ultrasound-guided injection and occlusion of the trachea in fetal sheep. Ultrasound Obstet Gynecol 28 (2006) 82-88
    • (2006) Ultrasound Obstet Gynecol , vol.28 , pp. 82-88
    • David, A.L.1    Weisz, B.2    Gregory, L.3
  • 57
    • 27944470542 scopus 로고    scopus 로고
    • Gene therapy progress and prospects: fetal gene therapy - first proofs of concept - some adverse effects
    • Coutelle C., Themis M., Waddington S.N., et al. Gene therapy progress and prospects: fetal gene therapy - first proofs of concept - some adverse effects. Gene Ther 12 (2005) 1601-1607
    • (2005) Gene Ther , vol.12 , pp. 1601-1607
    • Coutelle, C.1    Themis, M.2    Waddington, S.N.3
  • 58
    • 0034577051 scopus 로고    scopus 로고
    • Gene transfer into the fetal primate: evidence for the secretion of transgene product
    • Larson J.E., Morrow S.L., Delcarpio J.B., et al. Gene transfer into the fetal primate: evidence for the secretion of transgene product. Mol Ther 2 (2000) 631-639
    • (2000) Mol Ther , vol.2 , pp. 631-639
    • Larson, J.E.1    Morrow, S.L.2    Delcarpio, J.B.3
  • 59
    • 0035668699 scopus 로고    scopus 로고
    • Lentiviral vector gene transfer into fetal rhesus monkeys (Macaca mulatta): lung-targeting approaches
    • Tarantal A.F., Lee C.I., Ekert J.E., et al. Lentiviral vector gene transfer into fetal rhesus monkeys (Macaca mulatta): lung-targeting approaches. Mol Ther 4 (2001) 614-621
    • (2001) Mol Ther , vol.4 , pp. 614-621
    • Tarantal, A.F.1    Lee, C.I.2    Ekert, J.E.3
  • 60
    • 0033784808 scopus 로고    scopus 로고
    • Normal development of human fetal hematopoiesis between eight and seventeen weeks' gestation
    • Pahal G.S., Jauniaux E., Kinnon C., et al. Normal development of human fetal hematopoiesis between eight and seventeen weeks' gestation. Am J Obstet Gynecol 183 (2000) 1029-1034
    • (2000) Am J Obstet Gynecol , vol.183 , pp. 1029-1034
    • Pahal, G.S.1    Jauniaux, E.2    Kinnon, C.3
  • 61
    • 0029784268 scopus 로고    scopus 로고
    • Human fetal gene therapy: moral and ethical questions
    • Fletcher J.C., and Richter G. Human fetal gene therapy: moral and ethical questions. Hum Gene Ther 7 (1996) 1605-1614
    • (1996) Hum Gene Ther , vol.7 , pp. 1605-1614
    • Fletcher, J.C.1    Richter, G.2
  • 62
    • 0034180256 scopus 로고    scopus 로고
    • Prenatal gene transfer; scientific, medical, and ethical issues
    • Recombinant DNA Advisory Committee. Prenatal gene transfer; scientific, medical, and ethical issues. Hum Gene Ther 11 (2000) 1211-1229
    • (2000) Hum Gene Ther , vol.11 , pp. 1211-1229
    • Recombinant DNA Advisory Committee1
  • 63
    • 0031672371 scopus 로고    scopus 로고
    • Modfication of development by the CFTR gene in utero
    • Morrow S.L., Larson J.E., Nelson S., et al. Modfication of development by the CFTR gene in utero. Mol Genet Metab 65 (1998) 203-212
    • (1998) Mol Genet Metab , vol.65 , pp. 203-212
    • Morrow, S.L.1    Larson, J.E.2    Nelson, S.3
  • 64
    • 0037448352 scopus 로고    scopus 로고
    • A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency
    • Hacein-Bey-Abina S., von Kalle C., Schmidt M., et al. A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N Engl J Med 348 (2003) 255-256
    • (2003) N Engl J Med , vol.348 , pp. 255-256
    • Hacein-Bey-Abina, S.1    von Kalle, C.2    Schmidt, M.3
  • 65
    • 0038772394 scopus 로고    scopus 로고
    • What next for human gene therapy?
    • Juengst E.T. What next for human gene therapy?. Br Med J 326 (2003) 1410-1411
    • (2003) Br Med J , vol.326 , pp. 1410-1411
    • Juengst, E.T.1
  • 66
    • 33645451483 scopus 로고    scopus 로고
    • Mutagenesis and oncogenesis by chromosomal insertion of gene transfer vectors
    • Baum C., Kustikova O., Modlich U., et al. Mutagenesis and oncogenesis by chromosomal insertion of gene transfer vectors. Hum Gene Ther 17 (2006) 253-263
    • (2006) Hum Gene Ther , vol.17 , pp. 253-263
    • Baum, C.1    Kustikova, O.2    Modlich, U.3
  • 67
    • 23844507993 scopus 로고    scopus 로고
    • Oncogenesis following delivery of a nonprimate lentiviral gene therapy vector to fetal and neonatal mice
    • Themis M., Waddington S.N., Schmidt M., et al. Oncogenesis following delivery of a nonprimate lentiviral gene therapy vector to fetal and neonatal mice. Mol Ther 12 (2005) 763-771
    • (2005) Mol Ther , vol.12 , pp. 763-771
    • Themis, M.1    Waddington, S.N.2    Schmidt, M.3
  • 68
    • 0033021870 scopus 로고    scopus 로고
    • In utero gene therapy
    • Billings P.R. In utero gene therapy. Nat Med 5 (1999) 255-256
    • (1999) Nat Med , vol.5 , pp. 255-256
    • Billings, P.R.1
  • 69
    • 0033959097 scopus 로고    scopus 로고
    • In utero transfer and expression of exogenous genes in sheep
    • Tran N.D., Porada C.D., Zhao Y., et al. In utero transfer and expression of exogenous genes in sheep. Exp Hematol 28 (2000) 17-30
    • (2000) Exp Hematol , vol.28 , pp. 17-30
    • Tran, N.D.1    Porada, C.D.2    Zhao, Y.3
  • 70
    • 0034981261 scopus 로고    scopus 로고
    • In utero delivery of adeno-associated viral vectors: intraperitoneal gene transfer produces long-term expression
    • Lipshutz G.S., Gruber C.A., Cao Y., et al. In utero delivery of adeno-associated viral vectors: intraperitoneal gene transfer produces long-term expression. Mol Ther 3 (2001) 284-292
    • (2001) Mol Ther , vol.3 , pp. 284-292
    • Lipshutz, G.S.1    Gruber, C.A.2    Cao, Y.3
  • 71
    • 0033091484 scopus 로고    scopus 로고
    • In utero gene therapy: the case for
    • Schneider H., and Coutelle C. In utero gene therapy: the case for. Nat Med 5 (1999) 256-257
    • (1999) Nat Med , vol.5 , pp. 256-257
    • Schneider, H.1    Coutelle, C.2
  • 72
    • 0036272667 scopus 로고    scopus 로고
    • On the scientific and ethical issues of fetal somatic gene therapy
    • Coutelle C., and Rodeck C. On the scientific and ethical issues of fetal somatic gene therapy. Gene Ther 9 (2002) 670-673
    • (2002) Gene Ther , vol.9 , pp. 670-673
    • Coutelle, C.1    Rodeck, C.2
  • 73
    • 0034193478 scopus 로고    scopus 로고
    • Limitations of informed consent for in utero gene transfer research: implications for investigators and institutional review boards
    • Burger I.M., and Wilfond B.S. Limitations of informed consent for in utero gene transfer research: implications for investigators and institutional review boards. Hum Gene Ther 11 (2000) 1057-1063
    • (2000) Hum Gene Ther , vol.11 , pp. 1057-1063
    • Burger, I.M.1    Wilfond, B.S.2
  • 74
    • 0036677704 scopus 로고    scopus 로고
    • Prenatal T-cell reconstitution after in utero transplantation with fetal liver cells in a patient with X-linked severe combined immunodeficiency
    • Westgren M., Ringden O., Bartmann P., et al. Prenatal T-cell reconstitution after in utero transplantation with fetal liver cells in a patient with X-linked severe combined immunodeficiency. Am J Obstet Gynecol 187 (2002) 475-482
    • (2002) Am J Obstet Gynecol , vol.187 , pp. 475-482
    • Westgren, M.1    Ringden, O.2    Bartmann, P.3
  • 75
    • 0034478002 scopus 로고    scopus 로고
    • Preimplantation genetic diagnosis: applications for molecular medicine
    • Wells D., and Delhanty J.D. Preimplantation genetic diagnosis: applications for molecular medicine. Trends Mol Med 7 (2001) 23-30
    • (2001) Trends Mol Med , vol.7 , pp. 23-30
    • Wells, D.1    Delhanty, J.D.2
  • 76
    • 0030892816 scopus 로고    scopus 로고
    • Preimplantation diagnosis and other reproductive options: attitudes of male and female carriers of recessive disorders
    • Snowdon C., and Green J.M. Preimplantation diagnosis and other reproductive options: attitudes of male and female carriers of recessive disorders. Hum Reprod 12 (1997) 341-350
    • (1997) Hum Reprod , vol.12 , pp. 341-350
    • Snowdon, C.1    Green, J.M.2
  • 77
    • 39049102434 scopus 로고    scopus 로고
    • Regulations not keeping up with developments in genetics, says poll
    • Brown P. Regulations not keeping up with developments in genetics, says poll. Br Med J 321 (2000) 1369
    • (2000) Br Med J , vol.321 , pp. 1369
    • Brown, P.1
  • 78
    • 17744383751 scopus 로고    scopus 로고
    • Transplantation of a fetus with paternal Thy-1(+)CD34(+) cells for chronic granulomatous disease
    • Muench M.O., Rae J., Barcena A., et al. Transplantation of a fetus with paternal Thy-1(+)CD34(+) cells for chronic granulomatous disease. Bone Marrow Transplant 27 (2001) 355-364
    • (2001) Bone Marrow Transplant , vol.27 , pp. 355-364
    • Muench, M.O.1    Rae, J.2    Barcena, A.3
  • 79
    • 0032720658 scopus 로고    scopus 로고
    • Induction of transplantation tolerance in humans using stem cell transplants prenatally or postnatally
    • Touraine J.L. Induction of transplantation tolerance in humans using stem cell transplants prenatally or postnatally. Transplant Proc 31 (1999) 2735-2737
    • (1999) Transplant Proc , vol.31 , pp. 2735-2737
    • Touraine, J.L.1
  • 80
    • 0032768176 scopus 로고    scopus 로고
    • Successful expression of b-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 b-galactosidase and factor IX transgenes in fetal and neonatal sheep after ultrasound-guided percutaneous adenovirus vector administration into the umbilical vein. Gene Ther 6 (1999) 1239-1248
    • (1999) Gene Ther , vol.6 , pp. 1239-1248
    • Themis, M.1    Schneider, H.2    Kiserud, T.3
  • 81
    • 0025350317 scopus 로고
    • Fetal blood sampling from the intrahepatic vein: analysis of safety and clinical experience with 214 procedures
    • Nicolini U., Nicolaidis P., Fisk N.M., et al. Fetal blood sampling from the intrahepatic vein: analysis of safety and clinical experience with 214 procedures. J Obstet Gynaecol 76 (1990) 47-53
    • (1990) J Obstet Gynaecol , vol.76 , pp. 47-53
    • Nicolini, U.1    Nicolaidis, P.2    Fisk, N.M.3
  • 82
    • 24644444255 scopus 로고    scopus 로고
    • Targeting the respiratory muscles of fetal sheep for prenatal gene therapy for Duchenne muscular dystrophy
    • Weisz B., David A.L., Gregory L.G., et al. Targeting the respiratory muscles of fetal sheep for prenatal gene therapy for Duchenne muscular dystrophy. Am J Obstet Gynecol 193 (2005) 1105-1109
    • (2005) Am J Obstet Gynecol , vol.193 , pp. 1105-1109
    • Weisz, B.1    David, A.L.2    Gregory, L.G.3
  • 83
    • 84971064941 scopus 로고
    • Ultrasound guided blood sampling from fetal sheep
    • Newnham J.P., Kelly R.W., Boyne P., et al. Ultrasound guided blood sampling from fetal sheep. Aust J Agric Res 40 (1989) 401-407
    • (1989) Aust J Agric Res , vol.40 , pp. 401-407
    • Newnham, J.P.1    Kelly, R.W.2    Boyne, P.3
  • 84
    • 0031708021 scopus 로고    scopus 로고
    • Intraheptatic vein fetal blood sampling: current role in prenatal diagnosis
    • Chinnaiya A., Venkat A., Dawn C., et al. Intraheptatic vein fetal blood sampling: current role in prenatal diagnosis. J Obstet Gynaecol Res 24 (1998) 239-246
    • (1998) J Obstet Gynaecol Res , vol.24 , pp. 239-246
    • Chinnaiya, A.1    Venkat, A.2    Dawn, C.3
  • 85
    • 0031736286 scopus 로고    scopus 로고
    • Ultrasound-guided gene therapy to hepatocytes in utero
    • Wang G., Williamson R., Mueller G., et al. Ultrasound-guided gene therapy to hepatocytes in utero. Fetal Diagn Ther 13 (1998) 197-205
    • (1998) Fetal Diagn Ther , vol.13 , pp. 197-205
    • Wang, G.1    Williamson, R.2    Mueller, G.3
  • 86
    • 0043163482 scopus 로고    scopus 로고
    • Percutaneous ultrasound-guided injection of the trachea in fetal sheep: a novel technique to target the fetal airways
    • David A.L., Peebles D.M., Gregory L., et al. Percutaneous ultrasound-guided injection of the trachea in fetal sheep: a novel technique to target the fetal airways. Fetal Diagn Ther 18 (2003) 385-390
    • (2003) Fetal Diagn Ther , vol.18 , pp. 385-390
    • David, A.L.1    Peebles, D.M.2    Gregory, L.3
  • 87
    • 9144222976 scopus 로고    scopus 로고
    • 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 L.G., David A., et al. 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 (2004) 70-78
    • (2004) Gene Ther , vol.11 , pp. 70-78
    • Peebles, D.1    Gregory, L.G.2    David, A.3
  • 88
    • 33746218856 scopus 로고    scopus 로고
    • Clinically applicable procedure for gene delivery to fetal gut by ultrasound-guided gastric injection: toward prenatal prevention of early-onset intestinal diseases
    • David A.L., Peebles D.M., Gregory L., et al. 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 (2006) 767-779
    • (2006) Hum Gene Ther , vol.17 , pp. 767-779
    • David, A.L.1    Peebles, D.M.2    Gregory, L.3


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