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Volumn 6, Issue 3, 2013, Pages 255-264

Combining gene therapy and fetal hemoglobin induction for treatment of β-thalassemia

Author keywords

thalassemia; erythroid progenitor cells; gene therapy; hemoglobin; high persistency of fetal hemoglobin; sickle cell anemia

Indexed keywords

HEMOGLOBIN BETA CHAIN; HEMOGLOBIN F; LENTIVIRUS VECTOR; RETROVIRUS VECTOR; THALIDOMIDE; TRANSCRIPTION FACTOR GATA 1;

EID: 84879400495     PISSN: 17474086     EISSN: 17474094     Source Type: Journal    
DOI: 10.1586/ehm.13.24     Document Type: Review
Times cited : (13)

References (118)
  • 1
    • 27644467092 scopus 로고    scopus 로고
    • Molecular diagnosis of inherited disorders: Lessons from hemoglobinopathies
    • Patrinos GP, Kollia P, Papadakis MN. Molecular diagnosis of inherited disorders: lessons from hemoglobinopathies. Hum. Mutat. 26(5), 399-412 (2005).
    • (2005) Hum. Mutat. , vol.26 , Issue.5 , pp. 399-412
    • Patrinos, G.P.1    Kollia, P.2    Papadakis, M.N.3
  • 2
    • 0037365343 scopus 로고    scopus 로고
    • Screening and genetic diagnosis of haemoglobin disorders
    • Old JM. Screening and genetic diagnosis of haemoglobin disorders. Blood Rev. 17(1), 43-53 (2003).
    • (2003) Blood Rev. , vol.17 , Issue.1 , pp. 43-53
    • Old, J.M.1
  • 4
    • 77953406829 scopus 로고    scopus 로고
    • Global burden, distribution and prevention of β-thalassemias and hemoglobin e disorders
    • Colah R, Gorakshakar A, Nadkarni A. Global burden, distribution and prevention of β-thalassemias and hemoglobin E disorders. Expert Rev. Hematol. 3(1), 103-117 (2010).
    • (2010) Expert Rev. Hematol. , vol.3 , Issue.1 , pp. 103-117
    • Colah, R.1    Gorakshakar, A.2    Nadkarni, A.3
  • 5
    • 33846005126 scopus 로고    scopus 로고
    • Molecular therapies in β-thalassaemia
    • Quek L, Thein SL. Molecular therapies in β-thalassaemia. Br. J. Haematol. 136(3), 353-365 (2007).
    • (2007) Br. J. Haematol. , vol.136 , Issue.3 , pp. 353-365
    • Quek, L.1    Thein, S.L.2
  • 6
    • 68849115226 scopus 로고    scopus 로고
    • An electronic infrastructure for research and treatment of the thalassemias and other hemoglobinopathies: The Euro-mediterranean ITHANET project
    • Lederer CW, Basak AN, Aydinok Y et al. An electronic infrastructure for research and treatment of the thalassemias and other hemoglobinopathies: the Euro-mediterranean ITHANET project. Hemoglobin 33(3), 163-176 (2009).
    • (2009) Hemoglobin , vol.33 , Issue.3 , pp. 163-176
    • Lederer, C.W.1    Basak, A.N.2    Aydinok, Y.3
  • 7
    • 33845966411 scopus 로고    scopus 로고
    • Medicinal chemistry of fetal hemoglobin inducers for treatment of β-thalassemia
    • Gambari R, Fibach E. Medicinal chemistry of fetal hemoglobin inducers for treatment of β-thalassemia. Curr. Med. Chem. 14(2), 199-212 (2007).
    • (2007) Curr. Med. Chem. , vol.14 , Issue.2 , pp. 199-212
    • Gambari, R.1    Fibach, E.2
  • 8
    • 67651015956 scopus 로고    scopus 로고
    • Fetal hemoglobin chemical inducers for treatment of hemoglobinopa-thies
    • Testa U. Fetal hemoglobin chemical inducers for treatment of hemoglobinopa-thies. Ann. Hematol. 88(6), 505-528 (2009).
    • (2009) Ann. Hematol. , vol.88 , Issue.6 , pp. 505-528
    • Testa, U.1
  • 9
    • 34547210176 scopus 로고    scopus 로고
    • Co-inheritance of α-and β-thalassaemia in mice ameliorates thalassaemic phenotype
    • Voon HP, Wardan H, Vadolas J. Co-inheritance of α-and β-thalassaemia in mice ameliorates thalassaemic phenotype. Blood Cells Mol. Dis. 39 (2), 184-188 (2007).
    • (2007) Blood Cells Mol. Dis. , vol.39 , Issue.2 , pp. 184-188
    • Voon, H.P.1    Wardan, H.2    Vadolas, J.3
  • 10
    • 57349129112 scopus 로고    scopus 로고
    • Controlling α-globin: A review of α-globin expression and its impact on β-thalassemia
    • Voon HP, Vadolas J. Controlling α-globin: a review of α-globin expression and its impact on β-thalassemia. Haematologica 93(12), 1868-1876 (2008).
    • (2008) Haematologica , vol.93 , Issue.12 , pp. 1868-1876
    • Voon, H.P.1    Vadolas, J.2
  • 11
    • 84879346295 scopus 로고    scopus 로고
    • Sicke cell disease and other hemoglobinopathies
    • Elsevier
    • Steinberg MH. Sicke cell disease and other hemoglobinopathies. In: Goldman's Cecil Medicine. Elsevier, 1066-1075 (2011).
    • (2011) Goldman's Cecil Medicine , pp. 1066-1075
    • Steinberg, M.H.1
  • 12
    • 0002282636 scopus 로고    scopus 로고
    • The thalassemias: Disorders of globin synthesis
    • Prchal KK, Lichtman MA, Kipps TJ, Seligsohn U (Eds). McGraw-Hill, NY, USA
    • Weatherall DJ. The thalassemias: disorders of globin synthesis. In: Williams Hematology. Prchal KK, Lichtman MA, Kipps TJ, Seligsohn U (Eds). McGraw-Hill, NY, USA (2010).
    • (2010) Williams Hematology
    • Weatherall, D.J.1
  • 13
    • 84875155964 scopus 로고    scopus 로고
    • Gene therapy for hemoglobinopathies: Progress and challenges
    • Dong A, Rivella S, Breda L. Gene therapy for hemoglobinopathies: progress and challenges. Transl. Res. 161(4), 293-306 (2013).
    • (2013) Transl. Res. , vol.161 , Issue.4 , pp. 293-306
    • Dong, A.1    Rivella, S.2    Breda, L.3
  • 15
    • 29744465830 scopus 로고    scopus 로고
    • Fertility and pregnancy in thalassemia major
    • Tuck SM. Fertility and pregnancy in thalassemia major. Ann. NY Acad. Sci. 1054, 300-307 (2005).
    • (2005) Ann. NY Acad. Sci. , vol.1054 , pp. 300-307
    • Tuck, S.M.1
  • 16
    • 80052331583 scopus 로고    scopus 로고
    • The multifactorial origin of growth failure in thalassaemia
    • Skordis N, Kyriakou A. The multifactorial origin of growth failure in thalassaemia. Pediatr. Endocrinol. Rev. 8(Suppl. 2), 271-277 (2011).
    • (2011) Pediatr. Endocrinol. Rev. , vol.8 , Issue.SUPPL. 2 , pp. 271-277
    • Skordis, N.1    Kyriakou, A.2
  • 17
    • 0036420133 scopus 로고    scopus 로고
    • Growth and puberty and its management in thalassaemia
    • Sanctis D. Growth and puberty and its management in thalassaemia. Horm. Res. 58(Suppl. 1), 72-79 (2002).
    • (2002) Horm. Res. , vol.58 , Issue.SUPPL. 1 , pp. 72-79
    • Sanctis, D.1
  • 18
    • 79959268119 scopus 로고    scopus 로고
    • Complications of thalassemia major and their treatment
    • Borgna-Pignatti C, Gamberini MR. Complications of thalassemia major and their treatment. Expert Rev. Hematol. 4(3), 353-366 (2011).
    • (2011) Expert Rev. Hematol. , vol.4 , Issue.3 , pp. 353-366
    • Borgna-Pignatti, C.1    Gamberini, M.R.2
  • 19
    • 0022409396 scopus 로고
    • α and β cell evaluation in patients with thalassaemia intermedia and iron overload
    • De Sanctis V, Gamberini MR, Borgatti L, Atti G, Vullo C, Bagni B. α and β cell evaluation in patients with thalassaemia intermedia and iron overload. Postgrad. Med. J. 61(721), 963-967 (1985).
    • (1985) Postgrad. Med. J. , vol.61 , Issue.721 , pp. 963-967
    • De Sanctis, V.1    Gamberini, M.R.2    Borgatti, L.3    Atti, G.4    Vullo, C.5    Bagni, B.6
  • 20
    • 74049109710 scopus 로고    scopus 로고
    • Noninvasive assessment of liver fbrosis in thalassaemia major patients by transient elastography (TE)-lack of interference by iron deposition
    • Di Marco V, Bronte F, Cabibi D et al. Noninvasive assessment of liver fbrosis in thalassaemia major patients by transient elastography (TE)-lack of interference by iron deposition. Br. J. Haematol. 148 (3), 476-479 (2010).
    • (2010) Br. J. Haematol. , vol.148 , Issue.3 , pp. 476-479
    • Di Marco, V.1    Bronte, F.2    Cabibi, D.3
  • 21
    • 74749088621 scopus 로고    scopus 로고
    • Update on thalassemia: Clinical care and complications
    • Cunningham MJ. Update on thalassemia: clinical care and complications. Hematol. Oncol. Clin. North Am. 24(1), 215-227 (2010).
    • (2010) Hematol. Oncol. Clin. North Am. , vol.24 , Issue.1 , pp. 215-227
    • Cunningham, M.J.1
  • 22
    • 38649131460 scopus 로고    scopus 로고
    • On the road to gene therapy for β-thalassemia and sickle cell anemia
    • Bank A. On the road to gene therapy for β-thalassemia and sickle cell anemia. Pediatr. Hematol. Oncol. 25(1), 1-4 (2008).
    • (2008) Pediatr. Hematol. Oncol. , vol.25 , Issue.1 , pp. 1-4
    • Bank, A.1
  • 23
    • 42149153870 scopus 로고    scopus 로고
    • Current status of globin gene therapy for the treatment of β-thalassaemia
    • Lisowski L, Sadelain M. Current status of globin gene therapy for the treatment of β-thalassaemia. Br. J. Haematol. 141(3), 335-345 (2008).
    • (2008) Br. J. Haematol. , vol.141 , Issue.3 , pp. 335-345
    • Lisowski, L.1    Sadelain, M.2
  • 25
    • 77956915835 scopus 로고    scopus 로고
    • Gene therapy: Targeting β-thalassaemia
    • Persons DA. Gene therapy: targeting β-thalassaemia. Nature 467(7313), 277-278 (2010).
    • (2010) Nature , vol.467 , Issue.7313 , pp. 277-278
    • Persons, D.A.1
  • 26
    • 78249246343 scopus 로고    scopus 로고
    • Hemoglobin gene therapy for β-thalassemia
    • Bank A. Hemoglobin gene therapy for β-thalassemia. Hematol. Oncol. Clin. North Am. 24(6), 1187-1201 (2010).
    • (2010) Hematol. Oncol. Clin. North Am. , vol.24 , Issue.6 , pp. 1187-1201
    • Bank, A.1
  • 27
    • 73949108653 scopus 로고    scopus 로고
    • Gene therapy for homozygous β-thalassemia. Is it a reality?
    • Boulad F, Rivière I, Sadelain M. Gene therapy for homozygous β-thalassemia. Is it a reality? Hemoglobin 33(Suppl. 1), S188-S196 (2009).
    • (2009) Hemoglobin , vol.33 , Issue.SUPPL. 1
    • Boulad, F.1    Rivière, I.2    Sadelain, M.3
  • 29
    • 42549157069 scopus 로고    scopus 로고
    • Optimization of recombinant adeno-associated viral vectors for human β-globin gene transfer and transgene expression
    • Maina N, Zhong L, Li X et al. Optimization of recombinant adeno-associated viral vectors for human β-globin gene transfer and transgene expression. Hum. Gene Ther. 19(4), 365-375 (2008).
    • (2008) Hum. Gene Ther. , vol.19 , Issue.4 , pp. 365-375
    • Maina, N.1    Zhong, L.2    Li, X.3
  • 30
    • 0025543247 scopus 로고
    • Retroviral vectors for the β-globin gene that demonstrate improved titer and expression
    • Gelinas R, Novak U. Retroviral vectors for the β-globin gene that demonstrate improved titer and expression. Ann. NY Acad. Sci. 612, 427-441 (1990).
    • (1990) Ann. NY Acad. Sci. , vol.612 , pp. 427-441
    • Gelinas, R.1    Novak, U.2
  • 31
    • 0037682108 scopus 로고    scopus 로고
    • Evaluation of optimal expression cassette in retrovirus vector for β-thalassemia gene therapy
    • Dong WJ, Li B, Liu DP et al. Evaluation of optimal expression cassette in retrovirus vector for β-thalassemia gene therapy. Mol. Biotechnol. 24(2), 127-140 (2003).
    • (2003) Mol. Biotechnol. , vol.24 , Issue.2 , pp. 127-140
    • Dong, W.J.1    Li, B.2    Liu, D.P.3
  • 32
    • 79958181694 scopus 로고    scopus 로고
    • Current advances in retroviral gene therapy
    • Yi Y, Noh MJ, Lee KH. Current advances in retroviral gene therapy. Curr. Gene Ther. 11(3), 218-228 (2011).
    • (2011) Curr. Gene Ther. , vol.11 , Issue.3 , pp. 218-228
    • Yi, Y.1    Noh, M.J.2    Lee, K.H.3
  • 34
    • 13044309472 scopus 로고    scopus 로고
    • Stable transduction of quiescent CD34+CD38-human hematopoietic cells by HIV-1-based lentiviral vectors
    • Case SS, Price MA, Jordan CT et al. Stable transduction of quiescent CD34+CD38-human hematopoietic cells by HIV-1-based lentiviral vectors. Proc. Natl Acad. Sci. USA 96(6), 2988-2993 (1999).
    • (1999) Proc. Natl Acad. Sci. USA , vol.96 , Issue.6 , pp. 2988-2993
    • Case, S.S.1    Price, M.A.2    Jordan, C.T.3
  • 35
    • 39649110649 scopus 로고    scopus 로고
    • Locus control region elements HS1 and HS4 enhance the therapeutic effcacy of globin gene transfer in β-thalassemic mice
    • Lisowski L, Sadelain M. Locus control region elements HS1 and HS4 enhance the therapeutic effcacy of globin gene transfer in β-thalassemic mice. Blood 110(13), 4175-4178 (2007).
    • (2007) Blood , vol.110 , Issue.13 , pp. 4175-4178
    • Lisowski, L.1    Sadelain, M.2
  • 36
    • 39849094315 scopus 로고    scopus 로고
    • Globin lentiviral vector insertions can perturb the expression of endogenous genes in β-thalassemic hematopoietic cells
    • Hargrove PW, Kepes S, Hanawa H et al. Globin lentiviral vector insertions can perturb the expression of endogenous genes in β-thalassemic hematopoietic cells. Mol. Ther. 16(3), 525-533 (2008).
    • (2008) Mol. Ther. , vol.16 , Issue.3 , pp. 525-533
    • Hargrove, P.W.1    Kepes, S.2    Hanawa, H.3
  • 37
    • 33947099883 scopus 로고    scopus 로고
    • Site-specifc transfer of an intact β-globin gene cluster through a new targeting vector
    • Zhou HS, Zhao N, Li L et al. Site-specifc transfer of an intact β-globin gene cluster through a new targeting vector. Biochem. Biophys. Res. Commun. 356(1), 32-37 (2007).
    • (2007) Biochem. Biophys. Res. Commun. , vol.356 , Issue.1 , pp. 32-37
    • Zhou, H.S.1    Zhao, N.2    Li, L.3
  • 38
    • 4444356095 scopus 로고    scopus 로고
    • In vivo selection of genetically modifed erythroid cells using a jak2-based cell growth switch
    • Zhao S, Weinreich MA, Ihara K, Richard RE, Blau CA. In vivo selection of genetically modifed erythroid cells using a jak2-based cell growth switch. Mol. Ther. 10(3), 456-468 (2004).
    • (2004) Mol. Ther. , vol.10 , Issue.3 , pp. 456-468
    • Zhao, S.1    Weinreich, M.A.2    Ihara, K.3    Richard, R.E.4    Blau, C.A.5
  • 39
    • 77955867566 scopus 로고    scopus 로고
    • The challenge of obtaining therapeutic levels of genetically modifed hematopoietic stem cells in β-thalassemia patients
    • Persons DA. The challenge of obtaining therapeutic levels of genetically modifed hematopoietic stem cells in β-thalassemia patients. Ann. NY Acad. Sci. 1202, 69-74 (2010).
    • (2010) Ann. NY Acad. Sci. , vol.1202 , pp. 69-74
    • Persons, D.A.1
  • 40
    • 77949733300 scopus 로고    scopus 로고
    • Mobilization of hematopoietic stem cells in a thalassemic mouse model: Implications for human gene therapy of thalassemia
    • Yannaki E, Psatha N, Athanasiou E et al. Mobilization of hematopoietic stem cells in a thalassemic mouse model: implications for human gene therapy of thalassemia. Hum. Gene Ther. 21(3), 299-310 (2010).
    • (2010) Hum. Gene Ther. , vol.21 , Issue.3 , pp. 299-310
    • Yannaki, E.1    Psatha, N.2    Athanasiou, E.3
  • 41
    • 77955906593 scopus 로고    scopus 로고
    • Hematopoietic stem cell mobilization strategies for gene therapy of β thalassemia and sickle cell disease
    • Yannaki E, Stamatoyannopoulos G. Hematopoietic stem cell mobilization strategies for gene therapy of β thalassemia and sickle cell disease. Ann. NY Acad. Sci. 1202, 59-63 (2010).
    • (2010) Ann. NY Acad. Sci. , vol.1202 , pp. 59-63
    • Yannaki, E.1    Stamatoyannopoulos, G.2
  • 42
    • 79953023267 scopus 로고    scopus 로고
    • Optimization of lentiviral vectors generation for biomedical and clinical research purposes: Contemporary trends in technology development and applications
    • Kumar P, Woon-Khiong C. Optimization of lentiviral vectors generation for biomedical and clinical research purposes: contemporary trends in technology development and applications. Curr. Gene Ther. 11 (2), 144-153 (2011).
    • (2011) Curr. Gene Ther. , vol.11 , Issue.2 , pp. 144-153
    • Kumar, P.1    Woon-Khiong, C.2
  • 43
    • 45749135187 scopus 로고    scopus 로고
    • In vivo selection of genetically modifed erythro-blastic progenitors leads to long-term correction of β-thalassemia
    • Miccio A, Cesari R, Lotti F et al. In vivo selection of genetically modifed erythro-blastic progenitors leads to long-term correction of β-thalassemia. Proc. Natl Acad. Sci. USA 105(30), 10547-10552 (2008).
    • (2008) Proc. Natl Acad. Sci. USA , vol.105 , Issue.30 , pp. 10547-10552
    • Miccio, A.1    Cesari, R.2    Lotti, F.3
  • 44
    • 79956354395 scopus 로고    scopus 로고
    • Correction of murine β-thalassemia after minimal lentiviral gene transfer and homeostatic in vivo erythroid expansion
    • Negre O, Fusil F, Colomb C et al. Correction of murine β-thalassemia after minimal lentiviral gene transfer and homeostatic in vivo erythroid expansion. Blood 117(20), 5321-5331 (2011).
    • (2011) Blood , vol.117 , Issue.20 , pp. 5321-5331
    • Negre, O.1    Fusil, F.2    Colomb, C.3
  • 45
    • 0037772187 scopus 로고    scopus 로고
    • Successful treatment of murine β-thalassemia using in vivo selection of genetically modifed, drug-resistant hematopoietic stem cells
    • Persons DA, Allay ER, Sawai N et al. Successful treatment of murine β-thalassemia using in vivo selection of genetically modifed, drug-resistant hematopoietic stem cells. Blood 102(2), 506-513 (2003).
    • (2003) Blood , vol.102 , Issue.2 , pp. 506-513
    • Persons, D.A.1    Allay, E.R.2    Sawai, N.3
  • 46
    • 0037195091 scopus 로고    scopus 로고
    • Permanent and panerythroid correction of murine β thalassemia by multiple lentiviral integration in hematopoietic stem cells
    • Imren S, Payen E, Westerman KA et al. Permanent and panerythroid correction of murine β thalassemia by multiple lentiviral integration in hematopoietic stem cells. Proc. Natl Acad. Sci. USA 99(22), 14380-14385 (2002).
    • (2002) Proc. Natl Acad. Sci. USA , vol.99 , Issue.22 , pp. 14380-14385
    • Imren, S.1    Payen, E.2    Westerman, K.A.3
  • 47
    • 0343628721 scopus 로고    scopus 로고
    • Therapeutic haemoglobin synthesis in β-thalassaemic mice expressing lentivirus-encoded human β-globin
    • May C, Rivella S, Callegari J et al. Therapeutic haemoglobin synthesis in β-thalassaemic mice expressing lentivirus-encoded human β-globin. Nature 406(6791), 82-86 (2000).
    • (2000) Nature , vol.406 , Issue.6791 , pp. 82-86
    • May, C.1    Rivella, S.2    Callegari, J.3
  • 48
    • 0037085774 scopus 로고    scopus 로고
    • Successful treatment of murine β-thalassemia intermedia by transfer of the human β-globin gene
    • May C, Rivella S, Chadburn A, Sadelain M. Successful treatment of murine β-thalassemia intermedia by transfer of the human β-globin gene. Blood 99(6), 1902-1908 (2002).
    • (2002) Blood , vol.99 , Issue.6 , pp. 1902-1908
    • May, C.1    Rivella, S.2    Chadburn, A.3    Sadelain, M.4
  • 49
    • 9444281434 scopus 로고    scopus 로고
    • Successful correction of the human β-thalassemia major phenotype using a lentiviral vector
    • Puthenveetil G, Scholes J, Carbonell D et al. Successful correction of the human β-thalassemia major phenotype using a lentiviral vector. Blood 104 (12), 3445-3453 (2004).
    • (2004) Blood , vol.104 , Issue.12 , pp. 3445-3453
    • Puthenveetil, G.1    Scholes, J.2    Carbonell, D.3
  • 50
    • 79953117530 scopus 로고    scopus 로고
    • Therapeutic levels of fetal hemoglobin in erythroid progeny of β-thalassemic CD34+ cells after lentiviral vector-mediated gene transfer
    • Wilber A, Hargrove PW, Kim YS et al. Therapeutic levels of fetal hemoglobin in erythroid progeny of β-thalassemic CD34+ cells after lentiviral vector-mediated gene transfer. Blood 117(10), 2817-2826 (2011).
    • (2011) Blood , vol.117 , Issue.10 , pp. 2817-2826
    • Wilber, A.1    Hargrove, P.W.2    Kim, Y.S.3
  • 51
    • 9644291600 scopus 로고    scopus 로고
    • High-level β-globin expression and preferred intragenic integration after lentiviral transduction of human cord blood stem cells
    • Imren S, Fabry ME, Westerman KA et al. High-level β-globin expression and preferred intragenic integration after lentiviral transduction of human cord blood stem cells. J. Clin. Invest. 114 (7), 953-962 (2004).
    • (2004) J. Clin. Invest. , vol.114 , Issue.7 , pp. 953-962
    • Imren, S.1    Fabry, M.E.2    Westerman, K.A.3
  • 52
    • 0034783710 scopus 로고    scopus 로고
    • Toward gene therapy for disorders of globin synthesis
    • Tisdale J, Sadelain M. Toward gene therapy for disorders of globin synthesis. Semin. Hematol. 38(4), 382-392 (2001).
    • (2001) Semin. Hematol. , vol.38 , Issue.4 , pp. 382-392
    • Tisdale, J.1    Sadelain, M.2
  • 53
    • 0034890776 scopus 로고    scopus 로고
    • Lentivirus vectors: Diffculties and hopes before clinical trials
    • Kafri T. Lentivirus vectors: diffculties and hopes before clinical trials. Curr. Opin. Mol. Ther. 3(4), 316-326 (2001).
    • (2001) Curr. Opin. Mol. Ther. , vol.3 , Issue.4 , pp. 316-326
    • Kafri, T.1
  • 54
    • 70349185586 scopus 로고    scopus 로고
    • The 3′ region of the chicken hypersensitive site-4 insulator has properties similar to its core and is required for full insulator activity
    • Arumugam PI, Urbinati F, Velu CS, Higashimoto T, Grimes HL, Malik P. The 3′ region of the chicken hypersensitive site-4 insulator has properties similar to its core and is required for full insulator activity. PLoS ONE 4(9), e6995 (2009).
    • (2009) PLoS ONE , vol.4 , Issue.9
    • Arumugam, P.I.1    Urbinati, F.2    Velu, C.S.3    Higashimoto, T.4    Grimes, H.L.5    Malik, P.6
  • 55
    • 29744458522 scopus 로고    scopus 로고
    • A Phase I/II clinical trial of β-globin gene therapy for β-thalassemia
    • Bank A, Dorazio R, Leboulch P. A Phase I/II clinical trial of β-globin gene therapy for β-thalassemia. Ann. NY Acad. Sci. 1054, 308-316 (2005).
    • (2005) Ann. NY Acad. Sci. , vol.1054 , pp. 308-316
    • Bank, A.1    Dorazio, R.2    Leboulch, P.3
  • 56
    • 77955863845 scopus 로고    scopus 로고
    • A preclinical approach for gene therapy of β-thalassemia
    • Breda L, Kleinert DA, Casu C et al. A preclinical approach for gene therapy of β-thalassemia. Ann. NY Acad. Sci. 1202, 134-140 (2010).
    • (2010) Ann. NY Acad. Sci. , vol.1202 , pp. 134-140
    • Breda, L.1    Kleinert, D.A.2    Casu, C.3
  • 57
    • 77955904018 scopus 로고    scopus 로고
    • Strategy for a multicenter Phase i clinical trial to evaluate globin gene transfer in β-thalassemia
    • Sadelain M, Rivière I, Wang X et al. Strategy for a multicenter Phase I clinical trial to evaluate globin gene transfer in β-thalassemia. Ann. NY Acad. Sci. 1202, 52-58 (2010).
    • (2010) Ann. NY Acad. Sci. , vol.1202 , pp. 52-58
    • Sadelain, M.1    Rivière, I.2    Wang, X.3
  • 58
    • 0028401662 scopus 로고
    • Bone marrow transplantation in the treatment of thalassemia
    • Giardini C, Lucarelli G. Bone marrow transplantation in the treatment of thalassemia. Curr. Opin. Hematol. 1(2), 170-176 (1994).
    • (1994) Curr. Opin. Hematol. , vol.1 , Issue.2 , pp. 170-176
    • Giardini, C.1    Lucarelli, G.2
  • 59
    • 0024505741 scopus 로고
    • A majority of mice show long-term expression of a human β-globin gene after retrovirus transfer into hematopoietic stem cells
    • Bender MA, Gelinas RE, Miller AD. A majority of mice show long-term expression of a human β-globin gene after retrovirus transfer into hematopoietic stem cells. Mol. Cell. Biol. 9 (4), 1426-1434 (1989).
    • (1989) Mol. Cell. Biol. , vol.9 , Issue.4 , pp. 1426-1434
    • Bender, M.A.1    Gelinas, R.E.2    Miller, A.D.3
  • 60
    • 0023864651 scopus 로고
    • Lineage-specifc expression of a human β-globin gene in murine bone marrow transplant recipients reconstituted with retrovirus-transduced stem cells
    • Dzierzak EA, Papayannopoulou T, Mulligan RC. Lineage-specifc expression of a human β-globin gene in murine bone marrow transplant recipients reconstituted with retrovirus-transduced stem cells. Nature 331(6151), 35-41 (1988).
    • (1988) Nature , vol.331 , Issue.6151 , pp. 35-41
    • Dzierzak, E.A.1    Papayannopoulou, T.2    Mulligan, R.C.3
  • 61
    • 0023814416 scopus 로고
    • Expression of the human β-globin gene following retroviral-mediated transfer into multipotential hematopoietic progenitors of mice
    • Karlsson S, Bodine DM, Perry L, Papayannopoulou T, Nienhuis AW. Expression of the human β-globin gene following retroviral-mediated transfer into multipotential hematopoietic progenitors of mice. Proc. Natl Acad. Sci. USA 85(16), 6062-6066 (1988).
    • (1988) Proc. Natl Acad. Sci. USA , vol.85 , Issue.16 , pp. 6062-6066
    • Karlsson, S.1    Bodine, D.M.2    Perry, L.3    Papayannopoulou, T.4    Nienhuis, A.W.5
  • 62
    • 0034472742 scopus 로고    scopus 로고
    • In vivo silencing of the human γ-globin gene in murine erythroid cells following retroviral transduction
    • Lung HY, Meeus IS, Weinberg RS, Atweh GF. In vivo silencing of the human γ-globin gene in murine erythroid cells following retroviral transduction. Blood Cells Mol. Dis. 26 (6), 613-619 (2000).
    • (2000) Blood Cells Mol. Dis. , vol.26 , Issue.6 , pp. 613-619
    • Lung, H.Y.1    Meeus, I.S.2    Weinberg, R.S.3    Atweh, G.F.4
  • 63
    • 0026519349 scopus 로고
    • A 36-base-pair core sequence of locus control region enhances retrovirally transferred human β-globin gene expression
    • Chang JC, Liu D, Kan YW. A 36-base-pair core sequence of locus control region enhances retrovirally transferred human β-globin gene expression. Proc. Natl Acad. Sci. USA 89(7), 3107-3110 (1992).
    • (1992) Proc. Natl Acad. Sci. USA , vol.89 , Issue.7 , pp. 3107-3110
    • Chang, J.C.1    Liu, D.2    Kan, Y.W.3
  • 64
    • 0027534432 scopus 로고
    • A human β-globin gene fused to the human β-globin locus control region is expressed at high levels in erythroid cells of mice engrafted with retrovirus-transduced hematopoietic stem cells
    • Plavec I, Papayannopoulou T, Maury C, Meyer F. A human β-globin gene fused to the human β-globin locus control region is expressed at high levels in erythroid cells of mice engrafted with retrovirus-transduced hematopoietic stem cells. Blood 81(5), 1384-1392 (1993).
    • (1993) Blood , vol.81 , Issue.5 , pp. 1384-1392
    • Plavec, I.1    Papayannopoulou, T.2    Maury, C.3    Meyer, F.4
  • 65
    • 0033541517 scopus 로고    scopus 로고
    • Analysis of γ-globin expression cassettes in retrovirus vectors
    • Emery DW, Morrish F, Li Q, Stamatoyan-nopoulos G. Analysis of γ-globin expression cassettes in retrovirus vectors. Hum. Gene Ther. 10(6), 877-888 (1999).
    • (1999) Hum. Gene Ther. , vol.10 , Issue.6 , pp. 877-888
    • Emery, D.W.1    Morrish, F.2    Li, Q.3    Stamatoyan-Nopoulos, G.4
  • 66
    • 27544477087 scopus 로고    scopus 로고
    • Use of the hereditary persistence of fetal hemoglobin 2 enhancer to increase the expression of oncoretrovirus vectors for human y-globin
    • Fragkos M, Anagnou NP, Tubb J, Emery DW. Use of the hereditary persistence of fetal hemoglobin 2 enhancer to increase the expression of oncoretrovirus vectors for human y-globin. Gene Ther. 12(21), 1591-1600 (2005).
    • (2005) Gene Ther. , vol.12 , Issue.21 , pp. 1591-1600
    • Fragkos, M.1    Anagnou, N.P.2    Tubb, J.3    Emery, D.W.4
  • 67
    • 12944254524 scopus 로고    scopus 로고
    • Preselection of retrovirally transduced bone marrow avoids subsequent stem cell gene silencing and age-dependent extinction of expression of human ß-globin in engrafted mice
    • Kalberer CP, Pawliuk R, Imren S et al. Preselection of retrovirally transduced bone marrow avoids subsequent stem cell gene silencing and age-dependent extinction of expression of human ß-globin in engrafted mice. Proc. Natl Acad. Sci. USA 97(10), 5411-5415 (2000).
    • (2000) Proc. Natl Acad. Sci. USA , vol.97 , Issue.10 , pp. 5411-5415
    • Kalberer, C.P.1    Pawliuk, R.2    Imren, S.3
  • 69
    • 0037606048 scopus 로고    scopus 로고
    • A novel murine model of Cooley anemia and its rescue by lentiviral-mediated human ß-globin gene transfer
    • Rivella S, May C, Chadburn A, Rivière I, Sadelain M. A novel murine model of Cooley anemia and its rescue by lentiviral-mediated human ß-globin gene transfer. Blood 101(8), 2932-2939 (2003).
    • (2003) Blood , vol.101 , Issue.8 , pp. 2932-2939
    • Rivella, S.1    May, C.2    Chadburn, A.3    Rivière, I.4    Sadelain, M.5
  • 70
    • 0035861452 scopus 로고    scopus 로고
    • Correction of sickle cell disease in transgenic mouse models by gene therapy
    • Pawliuk R, Westerman KA, Fabry ME et al. Correction of sickle cell disease in transgenic mouse models by gene therapy. Science 294(5550), 2368-2371 (2001).
    • (2001) Science , vol.294 , Issue.5550 , pp. 2368-2371
    • Pawliuk, R.1    Westerman, K.A.2    Fabry, M.E.3
  • 71
    • 30544436003 scopus 로고    scopus 로고
    • A genetic strategy to treat sickle cell anemia by coregulating globin transgene expression and RNA interference
    • Samakoglu S, Lisowski L, Budak-Alpdogan T et al. A genetic strategy to treat sickle cell anemia by coregulating globin transgene expression and RNA interference. Nat. Biotechnol. 24(1), 89-94 (2006).
    • (2006) Nat. Biotechnol. , vol.24 , Issue.1 , pp. 89-94
    • Samakoglu, S.1    Lisowski, L.2    Budak-Alpdogan, T.3
  • 72
    • 82555187500 scopus 로고    scopus 로고
    • The GATA1-HS2 enhancer allows persistent and position-independent expression of a ß-globin transgene
    • Miccio A, Poletti V, Tiboni F et al. The GATA1-HS2 enhancer allows persistent and position-independent expression of a ß-globin transgene. PLoS ONE 6(12), e27955 (2011).
    • (2011) PLoS ONE , vol.6 , Issue.12
    • Miccio, A.1    Poletti, V.2    Tiboni, F.3
  • 73
    • 84858978712 scopus 로고    scopus 로고
    • Therapeutic hemoglobin levels after gene transfer in ß-thalassemia mice and in hematopoietic cells of ß-thalassemia and sickle cells disease patients
    • Breda L, Casu C, Gardenghi S et al Therapeutic hemoglobin levels after gene transfer in ß-thalassemia mice and in hematopoietic cells of ß-thalassemia and sickle cells disease patients. PLoS ONE 7(3), e32345 (2012).
    • (2012) PLoS ONE , vol.7 , Issue.3
    • Breda, L.1    Casu, C.2    Gardenghi, S.3
  • 74
    • 70449127224 scopus 로고    scopus 로고
    • Genotoxic potential of lineage-specifc lentivirus vectors carrying the ß-globin locus control region
    • Arumugam PI, Higashimoto T, Urbinati F et al. Genotoxic potential of lineage-specifc lentivirus vectors carrying the ß-globin locus control region. Mol. Ther. 17(11), 1929-1937 (2009).
    • (2009) Mol. Ther. , vol.17 , Issue.11 , pp. 1929-1937
    • Arumugam, P.I.1    Higashimoto, T.2    Urbinati, F.3
  • 75
    • 0034000919 scopus 로고    scopus 로고
    • The cHS4 insulator increases the probability of retroviral expression at random chromosomal integration sites
    • Rivella S, Callegari JA, May C, Tan CW, Sadelain M. The cHS4 insulator increases the probability of retroviral expression at random chromosomal integration sites. J. Virol. 74(10), 4679-4687 (2000).
    • (2000) J. Virol. , vol.74 , Issue.10 , pp. 4679-4687
    • Rivella, S.1    Callegari, J.A.2    May, C.3    Tan, C.W.4    Sadelain, M.5
  • 76
    • 79954994720 scopus 로고    scopus 로고
    • Genetic therapy for ß-thalassemia: From the bench to the bedside
    • Arumugam P, Malik P. Genetic therapy for ß-thalassemia: from the bench to the bedside. Hematology Am. Soc. Hematol. Educ. Program 2010, 445-450 (2010).
    • (2010) Hematology Am. Soc. Hematol. Educ. Program , vol.2010 , pp. 445-450
    • Arumugam, P.1    Malik, P.2
  • 77
    • 77956928344 scopus 로고    scopus 로고
    • Transfusion independence and HMGA2 activation after gene therapy of human ß-thalassaemia
    • Cavazzana-Calvo M, Payen E, Negre O et al. Transfusion independence and HMGA2 activation after gene therapy of human ß-thalassaemia. Nature 467(7313), 318-322 (2010).
    • (2010) Nature , vol.467 , Issue.7313 , pp. 318-322
    • Cavazzana-Calvo, M.1    Payen, E.2    Negre, O.3
  • 78
    • 72149123930 scopus 로고    scopus 로고
    • Gene therapy. β-thalassemia treatment succeeds, with a caveat
    • Kaiser J. Gene therapy. β-thalassemia treatment succeeds, with a caveat. Science 326 (5959), 1468-1469 (2009).
    • (2009) Science , vol.326 , Issue.5959 , pp. 1468-1469
    • Kaiser, J.1
  • 79
    • 79959935594 scopus 로고    scopus 로고
    • Distribution of lentiviral vector integration sites in mice following therapeutic gene transfer to treat β-thalassemia
    • Ronen K, Negre O, Roth S et al. Distribution of lentiviral vector integration sites in mice following therapeutic gene transfer to treat β-thalassemia. Mol. Ther. 19 (7), 1273-1286 (2011).
    • (2011) Mol. Ther. , vol.19 , Issue.7 , pp. 1273-1286
    • Ronen, K.1    Negre, O.2    Roth, S.3
  • 80
    • 77149153067 scopus 로고    scopus 로고
    • β-thalassemia
    • Cao A, Galanello R. β-thalassemia. Genet. Med. 12(2), 61-76 (2010).
    • (2010) Genet. Med. , vol.12 , Issue.2 , pp. 61-76
    • Cao, A.1    Galanello, R.2
  • 81
    • 83155167192 scopus 로고    scopus 로고
    • Recent advances in β-thalassemias
    • Cao A, Moi P, Galanello R. Recent advances in β-thalassemias. Pediatr. Rep. 3(2), e17 (2011).
    • (2011) Pediatr. Rep. , vol.3 , Issue.2
    • Cao, A.1    Moi, P.2    Galanello, R.3
  • 82
    • 74549128104 scopus 로고    scopus 로고
    • Foetal haemoglobin inducers and thalassaemia: Novel achievements
    • Gambari R. Foetal haemoglobin inducers and thalassaemia: novel achievements. Blood Transfus. 8(1), 5-7 (2010).
    • (2010) Blood Transfus. , vol.8 , Issue.1 , pp. 5-7
    • Gambari, R.1
  • 83
    • 70349496094 scopus 로고    scopus 로고
    • Fetal hemoglobin inducers from the natural world: A novel approach for identifcation of drugs for the treatment of β-thalassemia and sickle-cell anemia
    • Bianchi N, Zuccato C, Lampronti I, Borgatti M, Gambari R. Fetal hemoglobin inducers from the natural world: a novel approach for identifcation of drugs for the treatment of β-thalassemia and sickle-cell anemia. Evid. Based. Complement. Alternat. Med. 6(2), 141-151 (2009).
    • (2009) Evid. Based. Complement. Alternat. Med. , vol.6 , Issue.2 , pp. 141-151
    • Bianchi, N.1    Zuccato, C.2    Lampronti, I.3    Borgatti, M.4    Gambari, R.5
  • 84
    • 40549105388 scopus 로고    scopus 로고
    • A novel frameshift mutation (+A) at codon 18 of the β-globin gene associated with high persistence of fetal hemoglobin phenotype and Δβ-thalassemia
    • Feriotto G, Salvatori F, Finotti A et al. A novel frameshift mutation (+A) at codon 18 of the β-globin gene associated with high persistence of fetal hemoglobin phenotype and Δβ-thalassemia. Acta Haematol. 119(1), 28-37 (2008).
    • (2008) Acta Haematol. , vol.119 , Issue.1 , pp. 28-37
    • Feriotto, G.1    Salvatori, F.2    Finotti, A.3
  • 85
    • 67651097888 scopus 로고    scopus 로고
    • Amelioration of murine β-thalassemia through drug selection of hematopoietic stem cells transduced with a lentiviral vector encoding both γ-globin and the MGMT drug-resistance gene
    • Zhao H, Pestina TI, Nasimuzzaman M, Mehta P, Hargrove PW, Persons DA. Amelioration of murine β-thalassemia through drug selection of hematopoietic stem cells transduced with a lentiviral vector encoding both γ-globin and the MGMT drug-resistance gene. Blood 113(23), 5747-5756 (2009).
    • (2009) Blood , vol.113 , Issue.23 , pp. 5747-5756
    • Zhao, H.1    Pestina, T.I.2    Nasimuzzaman, M.3    Mehta, P.4    Hargrove, P.W.5    Persons, D.A.6
  • 86
    • 77957133346 scopus 로고    scopus 로고
    • Carrier screening for β-thalassaemia: A review of international practice
    • Cousens NE, Gaff CL, Metcalfe SA, Delatycki MB. Carrier screening for β-thalassaemia: a review of international practice. Eur. J. Hum. Genet. 18 (10), 1077-1083 (2010).
    • (2010) Eur. J. Hum. Genet. , vol.18 , Issue.10 , pp. 1077-1083
    • Cousens, N.E.1    Gaff, C.L.2    Metcalfe, S.A.3    Delatycki, M.B.4
  • 87
    • 38149059827 scopus 로고    scopus 로고
    • Pomalidomide and lenalidomide regulate erythropoiesis and fetal hemoglobin production in human CD34+ cells
    • Moutouh-De Parseval LA, Verhelle D, Glezer E et al. Pomalidomide and lenalidomide regulate erythropoiesis and fetal hemoglobin production in human CD34+ cells. J. Clin. Invest. 118(1), 248-258 (2008).
    • (2008) J. Clin. Invest. , vol.118 , Issue.1 , pp. 248-258
    • Moutouh-De Parseval, L.A.1    Verhelle, D.2    Glezer, E.3
  • 88
    • 79960843759 scopus 로고    scopus 로고
    • Novel therapeutic candidates, identifed by molecular modeling, induce γ-globin gene expression in vivo
    • Boosalis MS, Castaneda SA, Trudel M et al. Novel therapeutic candidates, identifed by molecular modeling, induce γ-globin gene expression in vivo. Blood Cells Mol. Dis. 47 (2), 107-116 (2011).
    • (2011) Blood Cells Mol. Dis. , vol.47 , Issue.2 , pp. 107-116
    • Boosalis, M.S.1    Castaneda, S.A.2    Trudel, M.3
  • 89
    • 67749088379 scopus 로고    scopus 로고
    • Response to hydroxyurea in β thalassemia major and intermedia: Experience in western India
    • Italia KY, Jijina FJ, Merchant R et al. Response to hydroxyurea in β thalassemia major and intermedia: experience in western India. Clin. Chim. Acta 407(1-2), 10-15 (2009).
    • (2009) Clin. Chim. Acta , vol.407 , Issue.1-2 , pp. 10-15
    • Italia, K.Y.1    Jijina, F.J.2    Merchant, R.3
  • 90
    • 71449113459 scopus 로고    scopus 로고
    • Hydroxyurea-induced hematological response in transfusion-independent β-thalassemia intermedia: Case series and review of literature
    • Ehsani MA, Hedayati-Asl AA, Bagheri A, Zeinali S, Rashidi A. Hydroxyurea-induced hematological response in transfusion-independent β-thalassemia intermedia: case series and review of literature. Pediatr. Hematol. Oncol. 26(8), 560-565 (2009).
    • (2009) Pediatr. Hematol. Oncol. , vol.26 , Issue.8 , pp. 560-565
    • Ehsani, M.A.1    Hedayati-Asl, A.A.2    Bagheri, A.3    Zeinali, S.4    Rashidi, A.5
  • 91
    • 74549203895 scopus 로고    scopus 로고
    • Optimal response to thalidomide in a patient with thalassaemia major resistant to conventional therapy
    • Masera N, Tavecchia L, Capra M et al. Optimal response to thalidomide in a patient with thalassaemia major resistant to conventional therapy. Blood Transfus. 8(1), 63-65 (2010).
    • (2010) Blood Transfus. , vol.8 , Issue.1 , pp. 63-65
    • Masera, N.1    Tavecchia, L.2    Capra, M.3
  • 93
    • 0037105522 scopus 로고    scopus 로고
    • Development of virus vectors for gene therapy of β chain hemoglobinopathies: Fanking with a chromatin insulator reduces γ-globin gene silencing in vivo
    • Emery DW, Yannaki E, Tubb J, Nishino T, Li Q, Stamatoyannopoulos G. Development of virus vectors for gene therapy of β chain hemoglobinopathies: fanking with a chromatin insulator reduces γ-globin gene silencing in vivo. Blood 100(6), 2012-2019 (2002).
    • (2002) Blood , vol.100 , Issue.6 , pp. 2012-2019
    • Emery, D.W.1    Yannaki, E.2    Tubb, J.3    Nishino, T.4    Li, Q.5    Stamatoyannopoulos, G.6
  • 94
    • 4944234406 scopus 로고    scopus 로고
    • Extended β-globin locus control region elements promote consistent therapeutic expression of a γ-globin lentiviral vector in murine β-thalassemia
    • Hanawa H, Hargrove PW, Kepes S, Srivastava DK, Nienhuis AW, Persons DA. Extended β-globin locus control region elements promote consistent therapeutic expression of a γ-globin lentiviral vector in murine β-thalassemia. Blood 104 (8), 2281-2290 (2004).
    • (2004) Blood , vol.104 , Issue.8 , pp. 2281-2290
    • Hanawa, H.1    Hargrove, P.W.2    Kepes, S.3    Srivastava, D.K.4    Nienhuis, A.W.5    Persons, D.A.6
  • 95
    • 0037443467 scopus 로고    scopus 로고
    • The degree of phenotypic correction of murine β-thalassemia intermedia following lentiviral-mediated transfer of a human γ-globin gene is infuenced by chromosomal position effects and vector copy number
    • Persons DA, Hargrove PW, Allay ER, Hanawa H, Nienhuis AW. The degree of phenotypic correction of murine β-thalassemia intermedia following lentiviral-mediated transfer of a human γ-globin gene is infuenced by chromosomal position effects and vector copy number. Blood 101(6), 2175-2183 (2003).
    • (2003) Blood , vol.101 , Issue.6 , pp. 2175-2183
    • Persons, D.A.1    Hargrove, P.W.2    Allay, E.R.3    Hanawa, H.4    Nienhuis, A.W.5
  • 96
    • 33745957856 scopus 로고    scopus 로고
    • Partial correction of murine β-thalassemia with a γ retrovirus vector for human γ-globin
    • Nishino T, Tubb J, Emery DW. Partial correction of murine β-thalassemia with a γ retrovirus vector for human γ-globin. Blood Cells Mol. Dis. 37(1), 1-7 (2006).
    • (2006) Blood Cells Mol. Dis. , vol.37 , Issue.1 , pp. 1-7
    • Nishino, T.1    Tubb, J.2    Emery, D.W.3
  • 98
    • 65349107708 scopus 로고    scopus 로고
    • Discovering the genetics underlying foetal haemoglobin production in adults
    • Thein SL, Menzel S. Discovering the genetics underlying foetal haemoglobin production in adults. Br. J. Haematol. 145(4), 455-467 (2009).
    • (2009) Br. J. Haematol. , vol.145 , Issue.4 , pp. 455-467
    • Thein, S.L.1    Menzel, S.2
  • 99
    • 80052500364 scopus 로고    scopus 로고
    • Progress in understanding the hemoglobin switch
    • Forget BG. Progress in understanding the hemoglobin switch. N. Engl. J. Med. 365(9), 852-854 (2011).
    • (2011) N. Engl. J. Med. , vol.365 , Issue.9 , pp. 852-854
    • Forget, B.G.1
  • 100
    • 80052439730 scopus 로고    scopus 로고
    • A functional element necessary for fetal hemoglobin silencing
    • Sankaran VG, Xu J, Byron R et al. A functional element necessary for fetal hemoglobin silencing. N. Engl. J. Med. 365(9), 807-814 (2011).
    • (2011) N. Engl. J. Med. , vol.365 , Issue.9 , pp. 807-814
    • Sankaran, V.G.1    Xu, J.2    Byron, R.3
  • 101
    • 84861350562 scopus 로고    scopus 로고
    • Recent advances in the molecular understanding of non-transfusion- dependent thalassemia
    • Galanello R. Recent advances in the molecular understanding of non-transfusion-dependent thalassemia. Blood Rev. 26(Suppl. 1), S7-S11 (2012).
    • (2012) Blood Rev. , vol.26 , Issue.SUPPL. 1
    • Galanello, R.1
  • 102
    • 67651167040 scopus 로고    scopus 로고
    • Induction of endogenous γ-globin gene expression with decoy oligonucleotide targeting Oct-1 transcription factor consensus sequence
    • Xu XS, Hong X, Wang G. Induction of endogenous γ-globin gene expression with decoy oligonucleotide targeting Oct-1 transcription factor consensus sequence. J. Hematol. Oncol. 2, 15 (2009).
    • (2009) J. Hematol. Oncol. , vol.2 , pp. 15
    • Xu, X.S.1    Hong, X.2    Wang, G.3
  • 103
    • 33746632097 scopus 로고    scopus 로고
    • CMYB is involved in the regulation of fetal hemoglobin production in adults
    • Jiang J, Best S, Menzel S et al. cMYB is involved in the regulation of fetal hemoglobin production in adults. Blood 108 (3), 1077-1083 (2006).
    • (2006) Blood , vol.108 , Issue.3 , pp. 1077-1083
    • Jiang, J.1    Best, S.2    Menzel, S.3
  • 104
    • 57849083996 scopus 로고    scopus 로고
    • Human fetal hemoglobin expression is regulated by the developmental stage-specifc repressor BCL11A
    • Sankaran VG, Menne TF, Xu J et al. Human fetal hemoglobin expression is regulated by the developmental stage-specifc repressor BCL11A. Science 322(5909), 1839-1842 (2008).
    • (2008) Science , vol.322 , Issue.5909 , pp. 1839-1842
    • Sankaran, V.G.1    Menne, T.F.2    Xu, J.3
  • 105
    • 77955866470 scopus 로고    scopus 로고
    • Transcrip-tional silencing of fetal hemoglobin by BCL11A
    • Sankaran VG, Xu J, Orkin SH. Transcrip-tional silencing of fetal hemoglobin by BCL11A. Ann. NY Acad. Sci. 1202, 64-68 (2010).
    • (2010) Ann. NY Acad. Sci. , vol.1202 , pp. 64-68
    • Sankaran, V.G.1    Xu, J.2    Orkin, S.H.3
  • 106
    • 84858660529 scopus 로고    scopus 로고
    • Targeted therapeutic strategies for fetal hemoglobin induction
    • Sankaran VG. Targeted therapeutic strategies for fetal hemoglobin induction. Hematology Am. Soc. Hematol. Educ. Program 2011, 459-465 (2011).
    • (2011) Hematology Am. Soc. Hematol. Educ. Program , vol.2011 , pp. 459-465
    • Sankaran, V.G.1
  • 107
    • 77955304167 scopus 로고    scopus 로고
    • The XmnI (G)γ polymorphism infuences hemoglobin F synthesis contrary to BCL11A and HBS1L-MYB SNPs in a cohort of 57 β-thalassemia intermedia patients
    • Nguyen TK, Joly P, Bardel C, Moulsma M, Bonello-Palot N, Francina A. The XmnI (G)γ polymorphism infuences hemoglobin F synthesis contrary to BCL11A and HBS1L-MYB SNPs in a cohort of 57 β-thalassemia intermedia patients. Blood Cells Mol. Dis. 45(2), 124-127 (2010).
    • (2010) Blood Cells Mol. Dis. , vol.45 , Issue.2 , pp. 124-127
    • Nguyen, T.K.1    Joly, P.2    Bardel, C.3    Moulsma, M.4    Bonello-Palot, N.5    Francina, A.6
  • 109
    • 79952169016 scopus 로고    scopus 로고
    • MicroRNA-15a and-16-1 act via MYB to elevate fetal hemoglobin expression in human trisomy 13
    • Sankaran VG, Menne TF, Scepanovic D et al. MicroRNA-15a and-16-1 act via MYB to elevate fetal hemoglobin expression in human trisomy 13. Proc. Natl Acad. Sci. USA 108 (4), 1519-1524 (2011).
    • (2011) Proc. Natl Acad. Sci. USA , vol.108 , Issue.4 , pp. 1519-1524
    • Sankaran, V.G.1    Menne, T.F.2    Scepanovic, D.3
  • 110
    • 77956630402 scopus 로고    scopus 로고
    • KLF1 regulates BCL11A expression and γ-to β-globin gene switching
    • Zhou D, Liu K, Sun CW, Pawlik KM, Townes TM. KLF1 regulates BCL11A expression and γ-to β-globin gene switching. Nat. Genet. 42 (9), 742-744 (2010).
    • (2010) Nat. Genet. , vol.42 , Issue.9 , pp. 742-744
    • Zhou, D.1    Liu, K.2    Sun, C.W.3    Pawlik, K.M.4    Townes, T.M.5
  • 111
    • 13544277695 scopus 로고    scopus 로고
    • Exploring strategies for the design of artifcial transcription factors: Targeting sites proximal to known regulatory regions for the induction of γ-globin expression and the treatment of sickle cell disease
    • Gräslund T, Li X, Magnenat L, Popkov M, Barbas CF 3rd. Exploring strategies for the design of artifcial transcription factors: targeting sites proximal to known regulatory regions for the induction of γ-globin expression and the treatment of sickle cell disease. J. Biol. Chem. 280(5), 3707-3714 (2005).
    • (2005) J. Biol. Chem. , vol.280 , Issue.5 , pp. 3707-3714
    • Gräslund, T.1    Li, X.2    Magnenat, L.3    Popkov, M.4    Barbas III, C.F.5
  • 112
    • 77951060091 scopus 로고    scopus 로고
    • A zinc-fnger transcriptional activator designed to interact with the γ-globin gene promoters enhances fetal hemoglobin production in primary human adult erythroblasts
    • Wilber A, Tschulena U, Hargrove PW et al. A zinc-fnger transcriptional activator designed to interact with the γ-globin gene promoters enhances fetal hemoglobin production in primary human adult erythroblasts. Blood 115(15), 3033-3041 (2010).
    • (2010) Blood , vol.115 , Issue.15 , pp. 3033-3041
    • Wilber, A.1    Tschulena, U.2    Hargrove, P.W.3
  • 113
    • 79953076380 scopus 로고    scopus 로고
    • Recombinant erythroid Kruppel-like factor fused to GATA1 up-regulates Δ-and γ-globin expression in erythroid cells
    • Zhu J, Chin K, Aerbajinai W, Trainor C, Gao P, Rodgers GP. Recombinant erythroid Kruppel-like factor fused to GATA1 up-regulates Δ-and γ-globin expression in erythroid cells. Blood 117 (11), 3045-3052 (2011).
    • (2011) Blood , vol.117 , Issue.11 , pp. 3045-3052
    • Zhu, J.1    Chin, K.2    Aerbajinai, W.3    Trainor, C.4    Gao, P.5    Rodgers, G.P.6
  • 114
    • 84855848552 scopus 로고    scopus 로고
    • Fetal hemoglobin levels and morbidity in untransfused patients with β-thalassemia intermedia
    • Musallam KM, Sankaran VG, Cappellini MD, Duca L, Nathan DG, Taher AT. Fetal hemoglobin levels and morbidity in untransfused patients with β-thalassemia intermedia. Blood 119(2), 364-367 (2012).
    • (2012) Blood , vol.119 , Issue.2 , pp. 364-367
    • Musallam, K.M.1    Sankaran, V.G.2    Cappellini, M.D.3    Duca, L.4    Nathan, D.G.5    Taher, A.T.6
  • 115
    • 84864982332 scopus 로고    scopus 로고
    • A combined approach for β-thalassemia based on gene therapy-mediated adult hemoglobin (HbA) production and fetal hemoglobin (HbF) induction
    • Zuccato C, Breda L, Salvatori F et al. A combined approach for β-thalassemia based on gene therapy-mediated adult hemoglobin (HbA) production and fetal hemoglobin (HbF) induction. Ann. Hematol. 91(8), 1201-1213 (2012).
    • (2012) Ann. Hematol. , vol.91 , Issue.8 , pp. 1201-1213
    • Zuccato, C.1    Breda, L.2    Salvatori, F.3
  • 116
    • 76449105802 scopus 로고    scopus 로고
    • Increase in γ-globin mRNA content in human erythroid cells treated with angelicin analogs
    • Lampronti I, Bianchi N, Zuccato C et al. Increase in γ-globin mRNA content in human erythroid cells treated with angelicin analogs. Int. J. Hematol. 90(3), 318-327 (2009).
    • (2009) Int. J. Hematol. , vol.90 , Issue.3 , pp. 318-327
    • Lampronti, I.1    Bianchi, N.2    Zuccato, C.3
  • 117
    • 79958007234 scopus 로고    scopus 로고
    • Induction of human fetal hemoglobin via the NRF2 antioxidant response signaling pathway
    • Macari ER, Lowrey CH. Induction of human fetal hemoglobin via the NRF2 antioxidant response signaling pathway. Blood 117(22), 5987-5997 (2011).
    • (2011) Blood , vol.117 , Issue.22 , pp. 5987-5997
    • MacAri, E.R.1    Lowrey, C.H.2
  • 118
    • 80052651626 scopus 로고    scopus 로고
    • Thalassemia Clinical Research Network. A pilot study of subcutaneous decitabine in β-thalassemia intermedia
    • Olivieri NF, Saunthararajah Y, Thayalasuthan V et al.; Thalassemia Clinical Research Network. A pilot study of subcutaneous decitabine in β-thalassemia intermedia. Blood 118 (10), 2708-2711 (2011).
    • (2011) Blood , vol.118 , Issue.10 , pp. 2708-2711
    • Olivieri, N.F.1    Saunthararajah, Y.2    Thayalasuthan, V.3


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