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Volumn 1, Issue 2, 2000, Pages 91-99

Gene therapy: Trials and tribulations

Author keywords

[No Author keywords available]

Indexed keywords

JESSE;

EID: 0034328925     PISSN: 14710056     EISSN: None     Source Type: Journal    
DOI: 10.1038/35038533     Document Type: Review
Times cited : (606)

References (77)
  • 1
    • 0033972869 scopus 로고    scopus 로고
    • Nonviral gene therapy: Promises and challenges
    • Li, S. & Huang, L Nonviral gene therapy: promises and challenges. Gene Ther. 7, 31-34 (2000).
    • (2000) Gene Ther. , vol.7 , pp. 31-34
    • Li, S.1    Huang, L.2
  • 2
    • 0004173503 scopus 로고    scopus 로고
    • Marcel Dekker, Inc., New York
    • Templeton, N. S. & Lasic, D. D. (eds) Gene Therapy: Therapeutic Mechanisms and Strategies (Marcel Dekker, Inc., New York, 2000). This book has several excellent chapters on viral and non-viral vectors written by experts in the field. It also describes a number of therapeutic approaches to diseases and requirements for regulatory affairs.
    • Gene Therapy: Therapeutic Mechanisms and Strategies , pp. 2000
    • Templeton, N.S.1    Lasic, D.D.2
  • 4
    • 0025039252 scopus 로고
    • Gene therapy
    • Verma, I. M. Gene therapy. Sci. Am. 263, 68-72 (1990).
    • (1990) Sci. Am. , vol.263 , pp. 68-72
    • Verma, I.M.1
  • 5
    • 0032580348 scopus 로고    scopus 로고
    • Human gene therapy
    • Anderson, W. F. Human gene therapy. Nature 392, 25-30 (1998).
    • (1998) Nature , vol.392 , pp. 25-30
    • Anderson, W.F.1
  • 6
    • 0027595949 scopus 로고
    • The basic science of gene therapy
    • Mulligan, R. C. The basic science of gene therapy. Science 260, 925-932 (1993).
    • (1993) Science , vol.260 , pp. 925-932
    • Mulligan, R.C.1
  • 7
    • 0026718123 scopus 로고
    • Human gene therapy comes of age
    • Miller, A. D. Human gene therapy comes of age. Nature 357, 455-460 (1992).
    • (1992) Nature , vol.357 , pp. 455-460
    • Miller, A.D.1
  • 8
    • 0023871422 scopus 로고
    • A safe packaging line for gene transfer: Separating viral genes on two different plasmids
    • Markowitz, D., Goff, S. & Bank, A. A safe packaging line for gene transfer: separating viral genes on two different plasmids. J. Virol. 62, 1120-1124 (1988).
    • (1988) J. Virol. , vol.62 , pp. 1120-1124
    • Markowitz, D.1    Goff, S.2    Bank, A.3
  • 9
    • 0342463159 scopus 로고
    • Safe and efficient generation of recombinant retroviruses with amphotropic and ecotropic host ranges
    • Danos, O. & Mulligan, R. C. Safe and efficient generation of recombinant retroviruses with amphotropic and ecotropic host ranges. Proc. Natl Acad. Sci. USA 85, 6460-6464 (1988).
    • (1988) Proc. Natl Acad. Sci. USA , vol.85 , pp. 6460-6464
    • Danos, O.1    Mulligan, R.C.2
  • 10
    • 0027228422 scopus 로고
    • Vesicular stomatitis virus G glycoprotein pseudotyped retroviral vectors: Concentration to very high titer and efficient gene transfer into mammalian and non-mammalian cells
    • Burns, J. C., Friedmann, T., Driever, W., Burrascano, M. & Yee, J. K. Vesicular stomatitis virus G glycoprotein pseudotyped retroviral vectors: concentration to very high titer and efficient gene transfer into mammalian and non-mammalian cells. Proc. Natl Acad. Sci. USA 90, 8033-8037 (1993). Use of vesicular stomatitis virus glycoprotein (VSVG) to replace the envelope protein in MLV vectors. The resulting chimeric virus, containing VSVG protein, is pantropic and can be concentrated by ultracentrifugation.
    • (1993) Proc. Natl Acad. Sci. USA , vol.90 , pp. 8033-8037
    • Burns, J.C.1    Friedmann, T.2    Driever, W.3    Burrascano, M.4    Yee, J.K.5
  • 11
    • 0346132122 scopus 로고
    • Self-inactivating retroviral vectors designed for transfer of whole genes into mammalian cells
    • Yu, S. F. et al. Self-inactivating retroviral vectors designed for transfer of whole genes into mammalian cells. Proc. Natl Acad. Sci. USA 83, 3194-3198 (1986).
    • (1986) Proc. Natl Acad. Sci. USA , vol.83 , pp. 3194-3198
    • Yu, S.F.1
  • 13
    • 1842387143 scopus 로고
    • An alternative approach to somatic cell gene therapy
    • St Louis, D. & Verma, I. M. An alternative approach to somatic cell gene therapy. Proc. Natl Acad. Sci. USA 85, 3150-3154 (1988). On transplantation of retroviralty-transduced mouse fibroblasts, producing and secreting human factor IX protein, the transcription of the transgene is 'shut off'.
    • (1988) Proc. Natl Acad. Sci. USA , vol.85 , pp. 3150-3154
    • St Louis, D.1    Verma, I.M.2
  • 14
    • 0025976935 scopus 로고
    • Genetically modified skin fibroblasts persist long after transplantation but gradually inactivate introduced genes
    • Palmer, T. D., Rosman, G. J., Osborne, W. R. & Miller, A. D. Genetically modified skin fibroblasts persist long after transplantation but gradually inactivate introduced genes. Proc. Natl Acad. Sci. USA 88, 1330-1334 (1991).
    • (1991) Proc. Natl Acad. Sci. USA , vol.88 , pp. 1330-1334
    • Palmer, T.D.1    Rosman, G.J.2    Osborne, W.R.3    Miller, A.D.4
  • 15
    • 0026654682 scopus 로고
    • Human immunodeficiency virus infection of cells arrested in the cell cycle
    • Lewis, P., Hensel, M. & Emerman, M. Human immunodeficiency virus infection of cells arrested in the cell cycle. EMBO J. 11, 3053-3053 (1992).
    • (1992) EMBO J. , vol.11 , pp. 3053-3053
    • Lewis, P.1    Hensel, M.2    Emerman, M.3
  • 16
    • 0027376309 scopus 로고
    • A nuclear localization signal within HIV-1 matrix protein that governs infection of non-dividing cells
    • Bukrinsky, M. I. et al. A nuclear localization signal within HIV-1 matrix protein that governs infection of non-dividing cells. Nature 365, 666-669 (1993).
    • (1993) Nature , vol.365 , pp. 666-669
    • Bukrinsky, M.I.1
  • 17
    • 0031711593 scopus 로고    scopus 로고
    • HIV-1: Fifteen proteins and an RNA
    • Frankel, A. D. & Young, J. A. HIV-1: fifteen proteins and an RNA. Annu. Rev. Biochem. 67, 1-25 (1998).
    • (1998) Annu. Rev. Biochem. , vol.67 , pp. 1-25
    • Frankel, A.D.1    Young, J.A.2
  • 18
    • 0029996147 scopus 로고    scopus 로고
    • In vivo gene delivery and stable transduction of non-dividing cells by a lentiviral vector
    • Naldini, L. et al. In vivo gene delivery and stable transduction of non-dividing cells by a lentiviral vector. Science 272, 263-267 (1996). The first use of a lentiviral vector to transduce genes in vitro and in vivo in non-dividing cells. The lentiviral vector was packaged in VSVG envelope protein to expand the host range.
    • (1996) Science , vol.272 , pp. 263-267
    • Naldini, L.1
  • 19
    • 0030716959 scopus 로고    scopus 로고
    • Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors
    • Kafri, T., Blomer, U., Peterson, D. A., Gage, F. H. & Verma, I. M. Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors. Nature Genet. 17, 314-317 (1997).
    • (1997) Nature Genet. , vol.17 , pp. 314-317
    • Kafri, T.1    Blomer, U.2    Peterson, D.A.3    Gage, F.H.4    Verma, I.M.5
  • 20
    • 0030819379 scopus 로고    scopus 로고
    • Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo
    • Zufferey, R., Nagy, D., Mandel, R. J., Naldini, L. & Trono, D. Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo. Nature Biotechnol. 15, 871-875 (1997).
    • (1997) Nature Biotechnol. , vol.15 , pp. 871-875
    • Zufferey, R.1    Nagy, D.2    Mandel, R.J.3    Naldini, L.4    Trono, D.5
  • 21
    • 0034646513 scopus 로고    scopus 로고
    • HIV-1 genome nuclear import is mediated by a central DNA flap
    • Zennou, V. et al. HIV-1 genome nuclear import is mediated by a central DNA flap. Cell 101, 173-185 (2000).
    • (2000) Cell , vol.101 , pp. 173-185
    • Zennou, V.1
  • 22
    • 0034040751 scopus 로고    scopus 로고
    • Gene transfer by lentiviral vectors is limited by nuclear translocation and rescued by HIV-1 pol sequences
    • Follenzi, A., Ailles, L. E., Bakovic, S., Geuna, M. & Naldini, L. Gene transfer by lentiviral vectors is limited by nuclear translocation and rescued by HIV-1 pol sequences. Nature Genet. 25, 217-222 (2000).
    • (2000) Nature Genet. , vol.25 , pp. 217-222
    • Follenzi, A.1    Ailles, L.E.2    Bakovic, S.3    Geuna, M.4    Naldini, L.5
  • 23
    • 0032978712 scopus 로고    scopus 로고
    • Woodchuck hepatitis virus posttranscriptional regulatory element enhances expression of transgenes delivered by retroviral vectors
    • Zufferey, R., Donello, J. E., Trono, D. & Hope, T. J. Woodchuck hepatitis virus posttranscriptional regulatory element enhances expression of transgenes delivered by retroviral vectors. J. Virol. 73, 2885-2892 (1999).
    • (1999) J. Virol. , vol.73 , pp. 2885-2892
    • Zufferey, R.1    Donello, J.E.2    Trono, D.3    Hope, T.J.4
  • 25
    • 0031743608 scopus 로고    scopus 로고
    • Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery
    • Zufferey, R. et al. Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery. J. Virol. 72, 9873-9880 (1998).
    • (1998) J. Virol. , vol.72 , pp. 9873-9880
    • Zufferey, R.1
  • 26
    • 0033029635 scopus 로고    scopus 로고
    • Minimum requirements for efficient transduction of dividing and nondividing cells by feline immunodeficiency virus vectors
    • Johnston, J. C. et al. Minimum requirements for efficient transduction of dividing and nondividing cells by feline immunodeficiency virus vectors. J. Virol. 73, 4991-5000 (1999).
    • (1999) J. Virol. , vol.73 , pp. 4991-5000
    • Johnston, J.C.1
  • 27
    • 0034628493 scopus 로고    scopus 로고
    • Development of a self-inactivating, minimal lentivirus vector based on simian immunodeficiency virus
    • Schnell, T., Foley, P., Wirth, M., Munch, J. & Uberla, K. Development of a self-inactivating, minimal lentivirus vector based on simian immunodeficiency virus. Hum. Gene Ther. 11, 439-447 (2000).
    • (2000) Hum. Gene Ther. , vol.11 , pp. 439-447
    • Schnell, T.1    Foley, P.2    Wirth, M.3    Munch, J.4    Uberla, K.5
  • 28
    • 0031953134 scopus 로고    scopus 로고
    • Efficient transduction of nondividing human cells by feline immunodeficiency virus lentiviral vectors
    • Poeschla, E. M., Wong-Staal, F. & Looney, D. J. Efficient transduction of nondividing human cells by feline immunodeficiency virus lentiviral vectors. Nature Med. 4, 354-357 (1998).
    • (1998) Nature Med. , vol.4 , pp. 354-357
    • Poeschla, E.M.1    Wong-Staal, F.2    Looney, D.J.3
  • 29
    • 0032757387 scopus 로고    scopus 로고
    • Stable gene transfer to the nervous system using a non-primate lentiviral vector
    • Mitrophanous, K. et al. Stable gene transfer to the nervous system using a non-primate lentiviral vector. Gene Ther. 6, 1808-1818 (1999).
    • (1999) Gene Ther. , vol.6 , pp. 1808-1818
    • Mitrophanous, K.1
  • 30
    • 0034219436 scopus 로고    scopus 로고
    • Adeno-associated viral vectors as gene delivery vehicles
    • Carter, P. J. & Samulski, R. J. Adeno-associated viral vectors as gene delivery vehicles. Int. J. Mol. Med. 6, 17-27 (2000).
    • (2000) Int. J. Mol. Med. , vol.6 , pp. 17-27
    • Carter, P.J.1    Samulski, R.J.2
  • 31
    • 0004250845 scopus 로고    scopus 로고
    • (eds Fields, B. N., Knipe, D. M. & Howley P. M.,) Lippincott-Raven, Philadelphia
    • Berns, K. I. in Fields Virology (eds Fields, B. N., Knipe, D. M. & Howley P. M.,) 2173-2198 (Lippincott-Raven, Philadelphia, 1996).
    • (1996) Fields Virology , pp. 2173-2198
    • Berns, K.I.1
  • 32
    • 0025214094 scopus 로고
    • Site-specific integration by adeno-associated virus
    • Kotin, R. M. et al. Site-specific integration by adeno-associated virus. Proc. Natl Acad. Sci. USA 87, 2211-2215 (1990).
    • (1990) Proc. Natl Acad. Sci. USA , vol.87 , pp. 2211-2215
    • Kotin, R.M.1
  • 33
    • 0032874343 scopus 로고    scopus 로고
    • High-fidelity correction of mutations at multiple chromosomal positions by adeno-asscciated virus vectors
    • Inoue, N., Hirata, R. K. & Russell, D. W. High-fidelity correction of mutations at multiple chromosomal positions by adeno-asscciated virus vectors. J. Virol. 73, 7376-7380 (1999).
    • (1999) J. Virol. , vol.73 , pp. 7376-7380
    • Inoue, N.1    Hirata, R.K.2    Russell, D.W.3
  • 34
    • 0031906147 scopus 로고    scopus 로고
    • Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions
    • Summerford, C. & Samulski, R. J. Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions. J. Virol. 72, 1438-1445 (1998).
    • (1998) J. Virol. , vol.72 , pp. 1438-1445
    • Summerford, C.1    Samulski, R.J.2
  • 35
    • 0032870158 scopus 로고    scopus 로고
    • Genetic capsid modifications allow efficient re-targeting of adeno-associated virus type 2
    • Girod, A. et al. Genetic capsid modifications allow efficient re-targeting of adeno-associated virus type 2. Nature Med. 5, 1052-1056 (1999).
    • (1999) Nature Med. , vol.5 , pp. 1052-1056
    • Girod, A.1
  • 36
    • 0031058475 scopus 로고    scopus 로고
    • Recombinant adeno-associated virus mediates a high level of gene transfer but less efficient integration in the K562 human hematopoietic cell line
    • Malik, P. et al. Recombinant adeno-associated virus mediates a high level of gene transfer but less efficient integration in the K562 human hematopoietic cell line. J. Virol. 71, 1776-1783 (1997).
    • (1997) J. Virol. , vol.71 , pp. 1776-1783
    • Malik, P.1
  • 37
    • 0034050904 scopus 로고    scopus 로고
    • Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector
    • Kay, M. A. et al. Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. Nature Genet. 24, 257-261 (2000). Gives the first hints of successful gene therapy in haemophilia B patients by using recombinant adeno-associated viral vectors. Expression of transduced factor IX could be detected in one patient.
    • (2000) Nature Genet. , vol.24 , pp. 257-261
    • Kay, M.A.1
  • 38
    • 0034612248 scopus 로고    scopus 로고
    • Transsplicing vectors expand the utility of adeno-associated virus for gene therapy
    • Yan, Z., Zhang, Y., Duan, D. & Engelhardt, J. F. Transsplicing vectors expand the utility of adeno-associated virus for gene therapy. Proc. Natl Acad. Sci. USA 97, 6716-6721 (2000).
    • (2000) Proc. Natl Acad. Sci. USA , vol.97 , pp. 6716-6721
    • Yan, Z.1    Zhang, Y.2    Duan, D.3    Engelhardt, J.F.4
  • 39
    • 0034033447 scopus 로고    scopus 로고
    • Increasing the size of rAAV-mediated expression cassettes in vivo by intermolecular joining of two complementary vectors
    • Nakai, H., Storm, T. A. & Kay, M. A. Increasing the size of rAAV-mediated expression cassettes in vivo by intermolecular joining of two complementary vectors. Nature Biotechnol. 18, 527-532 (2000).
    • (2000) Nature Biotechnol. , vol.18 , pp. 527-532
    • Nakai, H.1    Storm, T.A.2    Kay, M.A.3
  • 40
    • 0004250845 scopus 로고    scopus 로고
    • (eds Fields, B. N., Knipe D. M. & Howley, P. M.) Lippincott-Raven, Philadelphia
    • Shenk, T. in Fields Virology (eds Fields, B. N., Knipe D. M. & Howley, P. M.) 2111-2148 (Lippincott-Raven, Philadelphia, 1996).
    • (1996) Fields Virology , pp. 2111-2148
    • Shenk, T.1
  • 41
    • 0030752379 scopus 로고    scopus 로고
    • Advances in adenoviral vectors: From genetic engineering to their biology
    • Yeh, P. & Perricaudet, M. Advances in adenoviral vectors: from genetic engineering to their biology. FASEB J. 11, 615-623 (1997).
    • (1997) FASEB J. , vol.11 , pp. 615-623
    • Yeh, P.1    Perricaudet, M.2
  • 42
    • 0029943155 scopus 로고    scopus 로고
    • A new adenoviral vector: Replacement of all viral coding sequences with 28 kb of DNA independently expressing both full-length dystrophin and β-galactosidase
    • Kochanek, S. et al. A new adenoviral vector: Replacement of all viral coding sequences with 28 kb of DNA independently expressing both full-length dystrophin and β-galactosidase. Proc. Natl Acad. Sci. USA 93, 5731-5736 (1996).
    • (1996) Proc. Natl Acad. Sci. USA , vol.93 , pp. 5731-5736
    • Kochanek, S.1
  • 43
    • 0030462599 scopus 로고    scopus 로고
    • A helper-dependent adenovirus vector system: Removal of helper virus by Cre-mediated excision of the viral packaging signal
    • Parks, R. J. et al. A helper-dependent adenovirus vector system: removal of helper virus by Cre-mediated excision of the viral packaging signal. Proc. Natl Acad. Sci. USA 93, 13565-13570 (1996). References 42 and 43 describe the generation of new adenoviral, 'gutless', vectors in which all the viral genes required for viral propagation are provided in trans. The gutless vectors show long-term expression of the transgene.
    • (1996) Proc. Natl Acad. Sci. USA , vol.93 , pp. 13565-13570
    • Parks, R.J.1
  • 44
    • 13044304200 scopus 로고    scopus 로고
    • Administration of helper-dependent adenoviral vectors and sequential delivery of different vector serotype for long-term liver-directed gene transfer in baboons
    • Moral, N. et al. Administration of helper-dependent adenoviral vectors and sequential delivery of different vector serotype for long-term liver-directed gene transfer in baboons. Proc. Natl Acad. Sci. USA 96, 12816-12821 (1999).
    • (1999) Proc. Natl Acad. Sci. USA , vol.96 , pp. 12816-12821
    • Moral, N.1
  • 45
    • 0028934815 scopus 로고
    • Cellular and humoral immune responses to adenoviral vectors containing factor IX gene: Tolerization of factor IX and vector antigens allows for long-term expression
    • Dai, Y. et al. Cellular and humoral immune responses to adenoviral vectors containing factor IX gene: tolerization of factor IX and vector antigens allows for long-term expression. Proc. Natl Acad. Sci. USA 92, 1401-1405 (1995).
    • (1995) Proc. Natl Acad. Sci. USA , vol.92 , pp. 1401-1405
    • Dai, Y.1
  • 46
    • 0032530110 scopus 로고    scopus 로고
    • Cellular immune response to adenoviral vector infected cells does not require de novo viral gene expression: Implications for gene therapy
    • Kafri, T. et al. Cellular immune response to adenoviral vector infected cells does not require de novo viral gene expression: implications for gene therapy. Proc. Natl Acad. Sci. USA 95, 11377-11382 (1998). Even physically inactivated adenoviral particles can generate a cytotoxic T-cell response, raising concerns about adenoviral vectors as suitable tools for long-term gene therapy.
    • (1998) Proc. Natl Acad. Sci. USA , vol.95 , pp. 11377-11382
    • Kafri, T.1
  • 47
    • 0029936764 scopus 로고    scopus 로고
    • Immune responses to transgene-encoded proteins limit the stability of gene expression after injection of replication-defective adenovirus vectors
    • Tripathy, S. K., Black, H. B., Goldwasser, E. & Leiden, J. M. Immune responses to transgene-encoded proteins limit the stability of gene expression after injection of replication-defective adenovirus vectors. Nature Med. 2, 545-550 (1996).
    • (1996) Nature Med. , vol.2 , pp. 545-550
    • Tripathy, S.K.1    Black, H.B.2    Goldwasser, E.3    Leiden, J.M.4
  • 48
    • 0004250845 scopus 로고    scopus 로고
    • (eds Fields, B. N., Knipe D. M. & Howley, P. M.) Lippincott-Raven, Philadelphia
    • Wagner, R. R., & Rose, J. K. in Fields Virology (eds Fields, B. N., Knipe D. M. & Howley, P. M.) 1121-1136 (Lippincott-Raven, Philadelphia, 1996).
    • (1996) Fields Virology , pp. 1121-1136
    • Wagner, R.R.1    Rose, J.K.2
  • 49
    • 0033977047 scopus 로고    scopus 로고
    • Humoral immunity to adeno-associated virus type 2 vectors following administration to murine and nohhuman primate muscle
    • Chirmule, N. et al. Humoral immunity to adeno-associated virus type 2 vectors following administration to murine and nohhuman primate muscle. J. Virol. 74, 2420-2425 (2000).
    • (2000) J. Virol. , vol.74 , pp. 2420-2425
    • Chirmule, N.1
  • 50
    • 0033960160 scopus 로고    scopus 로고
    • Repeat transduction in the mouse lung by using adeno-associated virus vectors with different serotypes
    • Halbert, C. L., Rutledge, E. A., Allen, J. M., Russell, D. W. & Miller, A. D. Repeat transduction in the mouse lung by using adeno-associated virus vectors with different serotypes. J. Virol. 74, 1524-1532 (2000).
    • (2000) J. Virol. , vol.74 , pp. 1524-1532
    • Halbert, C.L.1    Rutledge, E.A.2    Allen, J.M.3    Russell, D.W.4    Miller, A.D.5
  • 51
    • 0034151775 scopus 로고    scopus 로고
    • Role of vector in activation of T cell subsets in immune responses against the secreted transgene product factor IX
    • Fields, P. A. et al. Role of vector in activation of T cell subsets in immune responses against the secreted transgene product factor IX. Mol. Ther. 1, 225-235 (2000).
    • (2000) Mol. Ther. , vol.1 , pp. 225-235
    • Fields, P.A.1
  • 52
    • 0034169676 scopus 로고    scopus 로고
    • Route of adminstration determines induction of T-cell-independent humoral responses to adeno-associated virus vectors
    • Xiao, W. et al. Route of adminstration determines induction of T-cell-independent humoral responses to adeno-associated virus vectors. Mol. Ther. 1, 323-329 (2000).
    • (2000) Mol. Ther. , vol.1 , pp. 323-329
    • Xiao, W.1
  • 53
    • 0033616680 scopus 로고    scopus 로고
    • Sustained correction of bleeding disorder in hemophilia B mice by gene therapy
    • C. R.
    • Wang, L. Takabe, K., Bidlingmaier, S. M., III, C. R. & Verma, I. M. Sustained correction of bleeding disorder in hemophilia B mice by gene therapy. Proc. Natl Acad. Sci. USA 96, 3906-3910 (1999).
    • (1999) Proc. Natl Acad. Sci. USA , vol.96 , pp. 3906-3910
    • Wang, L.1    Takabe, K.2    Bidlingmaier III, S.M.3    Verma, I.M.4
  • 55
    • 0034724857 scopus 로고    scopus 로고
    • Gene therapy of human severe combined immunodeficiency (SCID)-XI disease
    • Cavazzana-Calvo, M. et al. Gene therapy of human severe combined immunodeficiency (SCID)-XI disease. Science 288, 669-672 (2000). The first definitive example of successful gene therapy, in three children suffering from SCID-XI. The haematopoietic stem cells from the patients were transduced by recombinant retroviruses expressing the γc-subunit, which is common to many interleukin receptors.
    • (2000) Science , vol.288 , pp. 669-672
    • Cavazzana-Calvo, M.1
  • 56
    • 0031593688 scopus 로고    scopus 로고
    • + cells in ADA-deficient SCID neonates
    • + cells in ADA-deficient SCID neonates. Nature Med. 4, 775-780 (1998).
    • (1998) Nature Med. , vol.4 , pp. 775-780
    • Kohn, D.B.1
  • 57
    • 0028807727 scopus 로고
    • T lymphocyte-directed gene therapy for ADA-SCID: Initial trial results after 4 years
    • Blaese, R. M. et al. T lymphocyte-directed gene therapy for ADA-SCID: initial trial results after 4 years. Science 270, 475-480 (1995).
    • (1995) Science , vol.270 , pp. 475-480
    • Blaese, R.M.1
  • 58
    • 0028789792 scopus 로고
    • Gene therapy in peripheral blood lymphocytes and bone marrow for ADA-immunodeficient patients
    • Bordignon, C. et al. Gene therapy in peripheral blood lymphocytes and bone marrow for ADA-immunodeficient patients. Science 270, 470-475 (1995).
    • (1995) Science , vol.270 , pp. 470-475
    • Bordignon, C.1
  • 60
    • 0034199171 scopus 로고    scopus 로고
    • - bone marrow and cord-derived SCID-repopulating cells with third-generation lentiviral vectors
    • - bone marrow and cord-derived SCID-repopulating cells with third-generation lentiviral vectors. Mol. Ther. 1, 566-573 (2000). References 59 and 60 show successful long-term transduction of human haematopoietic stem cells by lentiviral vectors, without the use of agents such as growth factors and cytokines.
    • (2000) Mol. Ther. , vol.1 , pp. 566-573
    • Guenechea, G.1
  • 61
    • 0030997346 scopus 로고    scopus 로고
    • Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors
    • Snyder, R. O. et al. Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors. Nature Genet. 16, 270-276 (1997).
    • (1997) Nature Genet. , vol.16 , pp. 270-276
    • Snyder, R.O.1
  • 62
    • 0034137797 scopus 로고    scopus 로고
    • Sustained expression of therapeutic level of factor IX in hemophilia B dogs by AAV-mediated gene therapy in liver
    • Wang, L. Nichols, T. C., Read, M. S., Bellinger, D. A. & Verma, I. M. Sustained expression of therapeutic level of factor IX in hemophilia B dogs by AAV-mediated gene therapy in liver. Mol. Ther. 1, 154-158 (2000).
    • (2000) Mol. Ther. , vol.1 , pp. 154-158
    • Wang, L.1    Nichols, T.C.2    Read, M.S.3    Bellinger, D.A.4    Verma, I.M.5
  • 63
    • 0343628721 scopus 로고    scopus 로고
    • Therapeutic haemoglobin synthesis in thalassaemic mice expressing lentivirus-encoded humanglobin
    • May, C. et al. Therapeutic haemoglobin synthesis in thalassaemic mice expressing lentivirus-encoded humanglobin. Nature 406, 82-86 (2000).
    • (2000) Nature , vol.406 , pp. 82-86
    • May, C.1
  • 64
    • 0029857689 scopus 로고    scopus 로고
    • Targeting retrovirus entry
    • Cosset, F. L. & Russell, S. J. Targeting retrovirus entry. Gene Ther. 3, 946-956 (1996).
    • (1996) Gene Ther. , vol.3 , pp. 946-956
    • Cosset, F.L.1    Russell, S.J.2
  • 65
    • 0033954001 scopus 로고    scopus 로고
    • Targeting adenovirus
    • Wickham, T. J. Targeting adenovirus. Gene Ther. 7, 110-114 (2000).
    • (2000) Gene Ther. , vol.7 , pp. 110-114
    • Wickham, T.J.1
  • 66
    • 0033578320 scopus 로고    scopus 로고
    • Retroviral vectors preloaded with a viral receptor-ligand bridge protein are targeted to specific cell types
    • Boerger, A. L. Snitkovsky, S. & Young, J. A. Retroviral vectors preloaded with a viral receptor-ligand bridge protein are targeted to specific cell types. Proc. Natl Acad. Sci. USA 96, 9867-9872 (1999).
    • (1999) Proc. Natl Acad. Sci. USA , vol.96 , pp. 9867-9872
    • Boerger, A.L.1    Snitkovsky, S.2    Young, J.A.3
  • 67
    • 0033007934 scopus 로고    scopus 로고
    • Targeted adeno-associated virus vector transduction of nonpermissive cells mediated by a bispecific F(ab'γ)2 antibody
    • erratum 17, 393 (1999)
    • Bartlett, J. S., Kteinschmidt, J., Boucher, R. C. & Samulski, R. J. Targeted adeno-associated virus vector transduction of nonpermissive cells mediated by a bispecific F(ab'γ)2 antibody. Nature Biotechnol. 17, 181-186 (1999); erratum 17, 393 (1999).
    • (1999) Nature Biotechnol. , vol.17 , pp. 181-186
    • Bartlett, J.S.1    Kteinschmidt, J.2    Boucher, R.C.3    Samulski, R.J.4
  • 68
    • 0026720075 scopus 로고
    • Tight control of gene expression in mammalian cells by tetracycline-responsive promoters
    • Gossen, M. & Bujard, H. Tight control of gene expression in mammalian cells by tetracycline-responsive promoters. Proc. Natl Acad. Sci. USA 89, 5547-5551 (1992). First description of a regulable gene expression system that uses tetracycline. The transgene is 'turned off' in the presence of tetracycline and 'turned on' in its absence.
    • (1992) Proc. Natl Acad. Sci. USA , vol.89 , pp. 5547-5551
    • Gossen, M.1    Bujard, H.2
  • 69
    • 0034608802 scopus 로고    scopus 로고
    • Exploring the sequence space for tetracycline-dependent transcriptional activators: Novel mutations yield expanded range and sensitivity
    • Urlinger, S. et al. Exploring the sequence space for tetracycline-dependent transcriptional activators: Novel mutations yield expanded range and sensitivity. Proc. Natl Acad. Sci. USA 97, 7963-7968 (2000).
    • (2000) Proc. Natl Acad. Sci. USA , vol.97 , pp. 7963-7968
    • Urlinger, S.1
  • 70
    • 0029962802 scopus 로고    scopus 로고
    • Autonomous parvovirus transduction of a gene under control of tissue-specific or inducible promoters
    • Maxwell, I. H., Spitzer, A. L. Long, C. J. & Maxwell, F. Autonomous parvovirus transduction of a gene under control of tissue-specific or inducible promoters. Gene Ther. 3, 28-36 (1996).
    • (1996) Gene Ther. , vol.3 , pp. 28-36
    • Maxwell, I.H.1    Spitzer, A.L.2    Long, C.J.3    Maxwell, F.4
  • 71
    • 0034202721 scopus 로고    scopus 로고
    • Lentiviral vectors: Regulated gene expression
    • Kafri, T., Van Praag, H., Gage, F. H. & Verma, I. M. Lentiviral vectors: Regulated gene expression. Mol. Ther. 1, 516-521 (2000).
    • (2000) Mol. Ther. , vol.1 , pp. 516-521
    • Kafri, T.1    Van Praag, H.2    Gage, F.H.3    Verma, I.M.4
  • 73
    • 0029865151 scopus 로고    scopus 로고
    • Ecdysone-inducible gene expression in mammalian cells and transgenic mice
    • No, D., Yao, T. P. & Evans, R. M. Ecdysone-inducible gene expression in mammalian cells and transgenic mice. Proc. Natl Acad. Sci. USA 93, 3346-3351 (1996).
    • (1996) Proc. Natl Acad. Sci. USA , vol.93 , pp. 3346-3351
    • No, D.1    Yao, T.P.2    Evans, R.M.3
  • 74
    • 0032493446 scopus 로고    scopus 로고
    • High level transactivation by a modified Bombyx ecdysone receptor in mammalian cells without exogenous retinoid X receptor
    • Suhr, S. T., Gil, E. B., Senut, M. C. & Gage, F. H. High level transactivation by a modified Bombyx ecdysone receptor in mammalian cells without exogenous retinoid X receptor. Proc. Natl Acad. Sci. USA 95, 7999-8004 (1998).
    • (1998) Proc. Natl Acad. Sci. USA , vol.95 , pp. 7999-8004
    • Suhr, S.T.1    Gil, E.B.2    Senut, M.C.3    Gage, F.H.4
  • 75
    • 0027756895 scopus 로고
    • Controlling signal transduction with synthetic ligands
    • Spencer, D. M., Wandless, T. J., Schreiber, S. L. & Crabtree, G. R. Controlling signal transduction with synthetic ligands. Science 262, 1019-1024 (1993).
    • (1993) Science , vol.262 , pp. 1019-1024
    • Spencer, D.M.1    Wandless, T.J.2    Schreiber, S.L.3    Crabtree, G.R.4
  • 76
    • 0032949030 scopus 로고    scopus 로고
    • Regulated delivery of therapeutic proteins after in vivo somatic cell gene transfer
    • Ye, X. et al. Regulated delivery of therapeutic proteins after in vivo somatic cell gene transfer. Science 283, 88-91 (1999).
    • (1999) Science , vol.283 , pp. 88-91
    • Ye, X.1
  • 77
    • 0030931963 scopus 로고    scopus 로고
    • Stable and efficient gene transfer into the retina using an HIV-based lentiviral vector
    • Miyoshi, H., Takahashi, M., Gage, F. H. & Verma, I. M. Stable and efficient gene transfer into the retina using an HIV-based lentiviral vector. Proc. Natl Acad. Sci. USA 16, 10319-10323 (1997).
    • (1997) Proc. Natl Acad. Sci. USA , vol.16 , pp. 10319-10323
    • Miyoshi, H.1    Takahashi, M.2    Gage, F.H.3    Verma, I.M.4


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