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Volumn 98, Issue 2, 1997, Pages 247-253

Genetic treatment of the haemoglobinopathies: Recombinations and new combinations

Author keywords

Gene therapy; Globin gene expression; Retroviral vector; Sickle cell disease treatment; Thalassaemia treatment

Indexed keywords

BETA GLOBIN; CD34 ANTIGEN; GLOBIN; VIRUS VECTOR;

EID: 0030810446     PISSN: 00071048     EISSN: None     Source Type: Journal    
DOI: 10.1046/j.1365-2141.1997.2313048.x     Document Type: Review
Times cited : (24)

References (64)
  • 1
    • 0029056481 scopus 로고
    • Retroviral transduction and expression of the human alkyl transferase cDNA provides nitrosourea resistance to hematopoietic cells
    • Allay, J.A., Dumenco, L.L., Koc, O.N., Liu, L. & Gerson, S.L. (1995) Retroviral transduction and expression of the human alkyl transferase cDNA provides nitrosourea resistance to hematopoietic cells. Blood, 85, 3342-3351.
    • (1995) Blood , vol.85 , pp. 3342-3351
    • Allay, J.A.1    Dumenco, L.L.2    Koc, O.N.3    Liu, L.4    Gerson, S.L.5
  • 3
    • 0024505741 scopus 로고
    • A majority of mice show long-term expression of a human beta-globin gene after retrovirus transfer into hematopoietic stem cells
    • Bender, M.A., Gelinas, R.E. & Miller, A.D. (1989) A majority of mice show long-term expression of a human beta-globin gene after retrovirus transfer into hematopoietic stem cells. Molecular and Cellular Biology, 9, 1426-1434.
    • (1989) Molecular and Cellular Biology , vol.9 , pp. 1426-1434
    • Bender, M.A.1    Gelinas, R.E.2    Miller, A.D.3
  • 5
    • 0023904379 scopus 로고
    • Comparison of expression in hematopoietic cells by retroviral vectors carrying two genes
    • Bowtell, D.D., Cory, S., Johnson, G.R. & Gonda, T.J. (1988) Comparison of expression in hematopoietic cells by retroviral vectors carrying two genes. Journal of Virology, 62, 2464-2473.
    • (1988) Journal of Virology , vol.62 , pp. 2464-2473
    • Bowtell, D.D.1    Cory, S.2    Johnson, G.R.3    Gonda, T.J.4
  • 8
    • 0025089990 scopus 로고
    • Serial transplantation of methotrexate-resistant bone marrow: Protection of murine recipients from drug toxicity by progeny of transduced cells
    • Corey, C.A., DeSilva, A.D., Holland, C.A. & Williams, D.A. (1990) Serial transplantation of methotrexate-resistant bone marrow: protection of murine recipients from drug toxicity by progeny of transduced cells. Blood, 75, 337-343.
    • (1990) Blood , vol.75 , pp. 337-343
    • Corey, C.A.1    DeSilva, A.D.2    Holland, C.A.3    Williams, D.A.4
  • 9
    • 0029857689 scopus 로고    scopus 로고
    • Targeting retrovirus entry
    • Cosset, F.L. & Russell, S.J. (1996) Targeting retrovirus entry. Gene Therapy, 3, 946-956.
    • (1996) Gene Therapy , vol.3 , pp. 946-956
    • Cosset, F.L.1    Russell, S.J.2
  • 10
    • 0021136489 scopus 로고
    • Transcriptional interference in avian retroviruses: Implications for the promoter insertion model of leukaemogenesis
    • Cullen, B.R., Lomedico, P.T. & Ju, G. (1984) Transcriptional interference in avian retroviruses: implications for the promoter insertion model of leukaemogenesis. Nature, 307, 241-245.
    • (1984) Nature , vol.307 , pp. 241-245
    • Cullen, B.R.1    Lomedico, P.T.2    Ju, G.3
  • 12
    • 0023864651 scopus 로고
    • Lineage-specific expression of a human beta-globin gene in murine bone marrow transplant recipients reconstituted with retrovirus-transduced stem cells
    • Dzierzak, E., Papayopoulou, T. & Mulligan, R.C. (1988) Lineage-specific expression of a human beta-globin gene in murine bone marrow transplant recipients reconstituted with retrovirus-transduced stem cells. Nature, 331, 35-41.
    • (1988) Nature , vol.331 , pp. 35-41
    • Dzierzak, E.1    Papayopoulou, T.2    Mulligan, R.C.3
  • 13
    • 0029145011 scopus 로고
    • Regulated high-level human beta-globin gene expression in erythroid cells following recombinant adeno-associated virus-mediated gene transfer
    • Einerhand, M.P.W., Antoniou, M., Zolotukhin, S., Muzyczka, N., Berns, K.I., Grosveid, F. & Valerio, D. (1995) Regulated high-level human beta-globin gene expression in erythroid cells following recombinant adeno-associated virus-mediated gene transfer. Gene Therapy, 2, 336-343.
    • (1995) Gene Therapy , vol.2 , pp. 336-343
    • Einerhand, M.P.W.1    Antoniou, M.2    Zolotukhin, S.3    Muzyczka, N.4    Berns, K.I.5    Grosveid, F.6    Valerio, D.7
  • 14
    • 0027473809 scopus 로고
    • Synthetic human β-globin 5′HS2 constructs function as LCRs only in multicopy transgene concatamers
    • Ellis, J., Talbot, D., Dillon, N. & Grosveld, F. (1993) Synthetic human β-globin 5′HS2 constructs function as LCRs only in multicopy transgene concatamers. EMBO Journal, 12, 127-134.
    • (1993) EMBO Journal , vol.12 , pp. 127-134
    • Ellis, J.1    Talbot, D.2    Dillon, N.3    Grosveld, F.4
  • 15
    • 0022625565 scopus 로고
    • Quantitative analysis of gene suppression in integrated retrovirus vectors
    • Emerman, M. & Temin, H.M. (1986) Quantitative analysis of gene suppression in integrated retrovirus vectors. Molecular and Cellular Biology, 6, 792-800.
    • (1986) Molecular and Cellular Biology , vol.6 , pp. 792-800
    • Emerman, M.1    Temin, H.M.2
  • 17
    • 0028967307 scopus 로고
    • + peripheral blood progenitor cells after introduction of a mutated dihydrofolate reductase cDNA via retroviral gene transfer
    • + peripheral blood progenitor cells after introduction of a mutated dihydrofolate reductase cDNA via retroviral gene transfer. Blood, 85, 566-574.
    • (1995) Blood , vol.85 , pp. 566-574
    • Flasshove, M.1    Banerjee, D.2    Mineishi, S.3    Li, M.-X.4    Bertino, J.R.5    Moore, M.A.S.6
  • 18
    • 0027443269 scopus 로고
    • Germ-line transmission and developmental regulation of a 150-kb yeast artificial chromosome containing the human beta-globin locus in transgenic mice
    • Gaensler, K.M.L., Kitamura, M. & Kan, Y.W. (1993) Germ-line transmission and developmental regulation of a 150-kb yeast artificial chromosome containing the human beta-globin locus in transgenic mice. Proceedings of the National Academy of Sciences of the United States of America, 90, 11381-11385.
    • (1993) Proceedings of the National Academy of Sciences of the United States of America , vol.90 , pp. 11381-11385
    • Gaensler, K.M.L.1    Kitamura, M.2    Kan, Y.W.3
  • 21
    • 0023663887 scopus 로고
    • Position-independent, high-level expression of the human β-globin gene in transgenic mice
    • Grosveld, F., van Assendelft, G.B., Greaves, D.R. & Kollias, B. (1987) Position-independent, high-level expression of the human β-globin gene in transgenic mice. Cell, 51, 975-985.
    • (1987) Cell , vol.51 , pp. 975-985
    • Grosveld, F.1    Van Assendelft, G.B.2    Greaves, D.R.3    Kollias, B.4
  • 22
    • 0027490716 scopus 로고
    • Evidence that retroviruses integrate into post-replication host DNA
    • Hajihosseini, M., Iavachev, L. & Price, J. (1993) Evidence that retroviruses integrate into post-replication host DNA. EMBO Journal 12, 4969-4974.
    • (1993) EMBO Journal , vol.12 , pp. 4969-4974
    • Hajihosseini, M.1    Iavachev, L.2    Price, J.3
  • 24
    • 0029908260 scopus 로고    scopus 로고
    • Transduction of hematopoietic cells by foamy virus vectors
    • Hirata, R.K., Miller, A.D., Andrews, R.G. & Russell, D.W. (1996) Transduction of hematopoietic cells by foamy virus vectors. Blood, 88, 3654-3661.
    • (1996) Blood , vol.88 , pp. 3654-3661
    • Hirata, R.K.1    Miller, A.D.2    Andrews, R.G.3    Russell, D.W.4
  • 26
    • 0029896544 scopus 로고    scopus 로고
    • Tagging ribozyme reaction sites to follow trans-splicing in mammalian cells
    • Jones, J.T., Lee, S.-W. & Sullenger, B.A. (1996) Tagging ribozyme reaction sites to follow trans-splicing in mammalian cells. Nature Medicine, 2, 643-648.
    • (1996) Nature Medicine , vol.2 , pp. 643-648
    • Jones, J.T.1    Lee, S.-W.2    Sullenger, B.A.3
  • 29
    • 0028567517 scopus 로고
    • Tissue-specific targeting of retroviral vectors through ligand-receptor interactions
    • Kasahara, N., Dozy, A.M. & Kan, Y.W. (1994) Tissue-specific targeting of retroviral vectors through ligand-receptor interactions. Science, 266, 1373-1376.
    • (1994) Science , vol.266 , pp. 1373-1376
    • Kasahara, N.1    Dozy, A.M.2    Kan, Y.W.3
  • 31
    • 0025784145 scopus 로고
    • Transport of cationic amino acids by the mouse ectropic retrovirus receptor
    • Kim, J.W., Closs, E.I., Albritton, L.M. & Cunningham, J.M. (1991) Transport of cationic amino acids by the mouse ectropic retrovirus receptor. Nature, 352, 725-728.
    • (1991) Nature , vol.352 , pp. 725-728
    • Kim, J.W.1    Closs, E.I.2    Albritton, L.M.3    Cunningham, J.M.4
  • 32
    • 0028303758 scopus 로고
    • Mutagenesis of retroviral vectors transducing β-globin gene and β-globin locus control region derivates results in stable transmission of an active transcriptional structure
    • Leboulch, P., Huang, G.S., Humphries, R.K., Young, O.H., Eaves, C.J., Yuan, D.Y.H. & London, I.M. (1994) Mutagenesis of retroviral vectors transducing β-globin gene and β-globin locus control region derivates results in stable transmission of an active transcriptional structure. EMBO Journal, 13, 3065-3076.
    • (1994) EMBO Journal , vol.13 , pp. 3065-3076
    • Leboulch, P.1    Huang, G.S.2    Humphries, R.K.3    Young, O.H.4    Eaves, C.J.5    Yuan, D.Y.H.6    London, I.M.7
  • 34
    • 0024961622 scopus 로고
    • Sickle cell anaemia: A simple disease with no cure
    • Luzzatto, L. & Goodfellow, P. (1989) Sickle cell anaemia: a simple disease with no cure. Nature, 337, 17-18.
    • (1989) Nature , vol.337 , pp. 17-18
    • Luzzatto, L.1    Goodfellow, P.2
  • 35
    • 0030024868 scopus 로고    scopus 로고
    • Induction of cyclophosphamide-resistance by aldehyde dehydrogenase gene transfer
    • Magni, M., Shammah, S., Schiro, R., Mellado, W., Dalla-Favera, R. & Gianni, A.M. (1996) Induction of cyclophosphamide-resistance by aldehyde dehydrogenase gene transfer. Blood, 87, 1097-1103.
    • (1996) Blood , vol.87 , pp. 1097-1103
    • Magni, M.1    Shammah, S.2    Schiro, R.3    Mellado, W.4    Dalla-Favera, R.5    Gianni, A.M.6
  • 36
    • 0029163531 scopus 로고
    • Protection of mice from lethal doses of methotrexate by transplantation with transgenic marrow expressing drug-resistant DHFR activity
    • May, C.M., Gunther, R. & McIvor, R.S. (1995) Protection of mice from lethal doses of methotrexate by transplantation with transgenic marrow expressing drug-resistant DHFR activity. Blood, 86, 2439-2448.
    • (1995) Blood , vol.86 , pp. 2439-2448
    • May, C.M.1    Gunther, R.2    McIvor, R.S.3
  • 37
    • 0028062923 scopus 로고
    • Retroviral vector sequences inhibit human beta-globin gene expression in transgenic mice
    • McCune, S. & Townes, T.M. (1994) Retroviral vector sequences inhibit human beta-globin gene expression in transgenic mice. Nucleic Acids Research, 22, 4477-4481.
    • (1994) Nucleic Acids Research , vol.22 , pp. 4477-4481
    • McCune, S.1    Townes, T.M.2
  • 38
    • 0023767237 scopus 로고
    • Design of retrovirus vector for transfer and expression of the human beta-globin gene
    • Miller, A.D., Bender, M.A., Harris, E.A.S., Kaleko, M. & Gelinas, R.E. (1988) Design of retrovirus vector for transfer and expression of the human beta-globin gene. Journal of Virology, 62, 4337-4345.
    • (1988) Journal of Virology , vol.62 , pp. 4337-4345
    • Miller, A.D.1    Bender, M.A.2    Harris, E.A.S.3    Kaleko, M.4    Gelinas, R.E.5
  • 39
    • 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. & Miller, A.D. (1990) Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection. Molecular and Cellular Biology, 10, 4239-4292.
    • (1990) Molecular and Cellular Biology , vol.10 , pp. 4239-4292
    • Miller, D.G.1    Adam, M.A.2    Miller, A.D.3
  • 42
    • 0030226201 scopus 로고    scopus 로고
    • Resistance to cytosine arabinoside by retrovirally mediated gene transfer of human cytidine deaminase into murine fibroblast and hematopoietic cells
    • Momparler, R.L., Eliopoulos, N., Bovenzi, V., Letourneau, S., Greenbaum, M. & Cournoyer, D. (1996) Resistance to cytosine arabinoside by retrovirally mediated gene transfer of human cytidine deaminase into murine fibroblast and hematopoietic cells. Cancer Gene Therapy, 3, 331-338.
    • (1996) Cancer Gene Therapy , vol.3 , pp. 331-338
    • Momparler, R.L.1    Eliopoulos, N.2    Bovenzi, V.3    Letourneau, S.4    Greenbaum, M.5    Cournoyer, D.6
  • 43
    • 0029016285 scopus 로고
    • Retrovirus-mediated expression of a DNA repair protein in bone marrow protects hematopoietic cells from nitrosourea-induced toxicity in vitro and in vivo
    • Moritz, T., Mackay, W., Glassner, B.J., Williams, D.A. & Samson, L. (1995) Retrovirus-mediated expression of a DNA repair protein in bone marrow protects hematopoietic cells from nitrosourea-induced toxicity in vitro and in vivo. Cancer Research, 55, 2608-2614.
    • (1995) Cancer Research , vol.55 , pp. 2608-2614
    • Moritz, T.1    Mackay, W.2    Glassner, B.J.3    Williams, D.A.4    Samson, L.5
  • 44
    • 8544259164 scopus 로고    scopus 로고
    • In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector
    • Naldini, L., Blomer, U., Gallay, P., Ory, D., Mulligan, R., Gage, F.H., Verma, I.M. & Trono, D. (1996) In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science, 1282, 7929-7711.
    • (1996) Science , vol.1282 , pp. 7929-17711
    • Naldini, L.1    Blomer, U.2    Gallay, P.3    Ory, D.4    Mulligan, R.5    Gage, F.H.6    Verma, I.M.7    Trono, D.8
  • 45
    • 0029077347 scopus 로고
    • Regulation of globin expression in erythroid cells
    • Orkin, S.H. (1995) Regulation of globin expression in erythroid cells. European Journal of Biochemistry, 231, 271-281.
    • (1995) European Journal of Biochemistry , vol.231 , pp. 271-281
    • Orkin, S.H.1
  • 48
    • 0027534432 scopus 로고
    • A human beta-globin gene used to the human beta-globin locus control region is exposed at high levels in erythroid cells of mice engrafted with retrovirus transduced hematopoietic stem cells
    • Plavec, I., Papayannoopoulou, T., Maury, C. & Meyer, F. (1993) A human beta-globin gene used to the human beta-globin locus control region is exposed at high levels in erythroid cells of mice engrafted with retrovirus transduced hematopoietic stem cells. Blood, 81, 1384-1392.
    • (1993) Blood , vol.81 , pp. 1384-1392
    • Plavec, I.1    Papayannoopoulou, T.2    Maury, C.3    Meyer, F.4
  • 49
    • 0029164582 scopus 로고
    • Effects of retroviral vector design on expression of human adenosine deaminase in murine bone marrow transplant recipients engrafted with genetically modified cells
    • Riviere, I., Brose, K. & Mulligan, R.C. (1995) Effects of retroviral vector design on expression of human adenosine deaminase in murine bone marrow transplant recipients engrafted with genetically modified cells. Proceedings of the National Academy of Sciences of the United States of America, 92, 6733-6737.
    • (1995) Proceedings of the National Academy of Sciences of the United States of America , vol.92 , pp. 6733-6737
    • Riviere, I.1    Brose, K.2    Mulligan, R.C.3
  • 50
    • 0027158091 scopus 로고
    • Integration of murine leukemia virus DNA depends on mitosis
    • Roe, T.Y., Reynolds, T.C., Yu, G. & Brown, P.O. (1993) Integration of murine leukemia virus DNA depends on mitosis. EMBO Journal, 12, 2099-2108.
    • (1993) EMBO Journal , vol.12 , pp. 2099-2108
    • Roe, T.Y.1    Reynolds, T.C.2    Yu, G.3    Brown, P.O.4
  • 51
    • 0003128085 scopus 로고
    • Critical issues in the genetic treatment of the hemoglobinopathies
    • ed. by Y. Beuzard et al, INSERM/ J. Libbey Eurotext Ltd, Paris
    • Sadelain, M. (1995) Critical issues in the genetic treatment of the hemoglobinopathies. Sickle Cell Disease and Thalassaemias: New Trends in Therapy (ed. by Y. Beuzard et al), pp. 115-123. INSERM/ J. Libbey Eurotext Ltd, Paris.
    • (1995) Sickle Cell Disease and Thalassaemias: New Trends in Therapy , pp. 115-123
    • Sadelain, M.1
  • 53
    • 0024311525 scopus 로고
    • Helper-free stocks of recombinant adeno-associated viruses: Normal integration does not require viral gene expression
    • Samulski, R.J., Chang, L-S. & Shenk, T. (1989) Helper-free stocks of recombinant adeno-associated viruses: normal integration does not require viral gene expression. Journal of Virology, 63, 3822-2828.
    • (1989) Journal of Virology , vol.63 , pp. 3822-12828
    • Samulski, R.J.1    Chang, L.-S.2    Shenk, T.3
  • 55
    • 12644266362 scopus 로고    scopus 로고
    • Constitutive overexpression of the cytidine deaminase gene confers resistance to cytosine arabinoside in vitro
    • Schroder, J.K., Kirch, C., Flasshove, M., Kalweit, H., Seidelmann, M., Hilger, R., Seeber, S. & Schutte, J. (1996) Constitutive overexpression of the cytidine deaminase gene confers resistance to cytosine arabinoside in vitro. Leukemia, 10, 1919-1924.
    • (1996) Leukemia , vol.10 , pp. 1919-1924
    • Schroder, J.K.1    Kirch, C.2    Flasshove, M.3    Kalweit, H.4    Seidelmann, M.5    Hilger, R.6    Seeber, S.7    Schutte, J.8
  • 57
    • 0029865455 scopus 로고    scopus 로고
    • A gene transfer strategy for making bone marrow cells resistant to trimetrexate
    • Spencer, H.T., Sleep, S.E.H., Rehg, J.E., Blakley, J. & Sorrentino, B.P. (1996) A gene transfer strategy for making bone marrow cells resistant to trimetrexate. Blood, 87, 2579-2587.
    • (1996) Blood , vol.87 , pp. 2579-2587
    • Spencer, H.T.1    Sleep, S.E.H.2    Rehg, J.E.3    Blakley, J.4    Sorrentino, B.P.5
  • 60
    • 0030059301 scopus 로고    scopus 로고
    • Mutagenesis in mammalian cells induced by triple helix formation and transcription-coupled repair
    • Wang, G.M., Seidman, M.M. & Glazer, P.M. (1996) Mutagenesis in mammalian cells induced by triple helix formation and transcription-coupled repair. Science, 271, 802-805.
    • (1996) Science , vol.271 , pp. 802-805
    • Wang, G.M.1    Seidman, M.M.2    Glazer, P.M.3
  • 61
    • 0025734320 scopus 로고
    • Cell-surface receptor for ecotropic murine retroviruses is a basic amino acid transporter
    • Wang, H., Kavanaugh, M.P., North, R.A. & Kabat, D. (1991) Cell-surface receptor for ecotropic murine retroviruses is a basic amino acid transporter. Nature, 352, 729-731.
    • (1991) Nature , vol.352 , pp. 729-731
    • Wang, H.1    Kavanaugh, M.P.2    North, R.A.3    Kabat, D.4
  • 64
    • 0028403232 scopus 로고
    • Long term protection of recipient mice from lethal doses of methotrexate by marrow transduced with a double copy retrovirus vector containing a mutated DHFR
    • Zhao, S.C., Li, M., Banerjee, D., Schweitzer, B.I., Mineishi, S., Gilboa, E. & Bertino, J.R. (1994) Long term protection of recipient mice from lethal doses of methotrexate by marrow transduced with a double copy retrovirus vector containing a mutated DHFR. Cancer Gene Therapy, 1, 27-33.
    • (1994) Cancer Gene Therapy , vol.1 , pp. 27-33
    • Zhao, S.C.1    Li, M.2    Banerjee, D.3    Schweitzer, B.I.4    Mineishi, S.5    Gilboa, E.6    Bertino, J.R.7


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