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Volumn 43, Issue 5, 2015, Pages 346-351

Potentially therapeutic levels of anti-sickling globin gene expression following lentivirus-mediated gene transfer in sickle cell disease bone marrow CD34+ cells

Author keywords

[No Author keywords available]

Indexed keywords

CD34 ANTIGEN; HEMOGLOBIN; HEMOGLOBIN F; HEMOGLOBIN GAMMA CHAIN; LENTIVIRUS VECTOR; MESSENGER RNA; HEMOGLOBIN BETA CHAIN;

EID: 84929047585     PISSN: 0301472X     EISSN: 18732399     Source Type: Journal    
DOI: 10.1016/j.exphem.2015.01.009     Document Type: Article
Times cited : (32)

References (21)
  • 1
    • 0030853711 scopus 로고    scopus 로고
    • Pathogenesis and treatment of sickle cell disease
    • Bunn H.F. Pathogenesis and treatment of sickle cell disease. N Engl J Med 1997, 337:762-769.
    • (1997) N Engl J Med , vol.337 , pp. 762-769
    • Bunn, H.F.1
  • 2
    • 33646419065 scopus 로고    scopus 로고
    • Stem cell therapy for sickle cell disease: Transplantation and gene therapy
    • Walters M.C. Stem cell therapy for sickle cell disease: Transplantation and gene therapy. Hematology Am Soc Hematol Educ Program 2005, 1:66-73.
    • (2005) Hematology Am Soc Hematol Educ Program , vol.1 , pp. 66-73
    • Walters, M.C.1
  • 3
    • 0016760198 scopus 로고
    • A new form of hereditary persistence of fetal hemoglobin in blacks and its association with sickle cell trait
    • Stamatoyannopoulos G., Wood W.G., Papayannopoulou T., Nute P.E. A new form of hereditary persistence of fetal hemoglobin in blacks and its association with sickle cell trait. Blood 1975, 46:683-692.
    • (1975) Blood , vol.46 , pp. 683-692
    • Stamatoyannopoulos, G.1    Wood, W.G.2    Papayannopoulou, T.3    Nute, P.E.4
  • 4
    • 0017367181 scopus 로고
    • Heterocellular hereditary persistence of fetal haemoglobin and homozygous sickle-cell disease
    • Serjeant G.R., Serjeant B.E., Mason K. Heterocellular hereditary persistence of fetal haemoglobin and homozygous sickle-cell disease. Lancet 1977, 1:795-796.
    • (1977) Lancet , vol.1 , pp. 795-796
    • Serjeant, G.R.1    Serjeant, B.E.2    Mason, K.3
  • 5
    • 0346497365 scopus 로고
    • Common haplotype dependency of high G gamma-globin gene expression and high HbF levels in beta-thalassemia and sickle cell anemia patients
    • Labie D., Pagnier J., Lapoumeroulie C., et al. Common haplotype dependency of high G gamma-globin gene expression and high HbF levels in beta-thalassemia and sickle cell anemia patients. Proc Natl Acad Sci U S A 1985, 82:2111-2114.
    • (1985) Proc Natl Acad Sci U S A , vol.82 , pp. 2111-2114
    • Labie, D.1    Pagnier, J.2    Lapoumeroulie, C.3
  • 6
    • 0003154471 scopus 로고    scopus 로고
    • The thalassemias
    • W.B. Saunders Company, Philadelphia, Penn, G. Stamatoyannopoulos, P. Majerus, R. Perlmutter, H.E. Varmus (Eds.)
    • Weatherall D.J. The thalassemias. The Molecular Basis for Blood Disorders 2001, 183-226. W.B. Saunders Company, Philadelphia, Penn. G. Stamatoyannopoulos, P. Majerus, R. Perlmutter, H.E. Varmus (Eds.).
    • (2001) The Molecular Basis for Blood Disorders , pp. 183-226
    • Weatherall, D.J.1
  • 7
    • 0021343093 scopus 로고
    • Is there a threshold level of fetal hemoglobin that ameliorates morbidity in sickle cell anemia?
    • Powars D.R., Weiss J.N., Chan L.S., Schroeder W.A. Is there a threshold level of fetal hemoglobin that ameliorates morbidity in sickle cell anemia?. Blood 1984, 63:921-926.
    • (1984) Blood , vol.63 , pp. 921-926
    • Powars, D.R.1    Weiss, J.N.2    Chan, L.S.3    Schroeder, W.A.4
  • 8
    • 0033560094 scopus 로고    scopus 로고
    • Induction of fetal hemoglobin in sickle cell disease
    • Bunn H.F. Induction of fetal hemoglobin in sickle cell disease. Blood 1999, 93:1787-1789.
    • (1999) Blood , vol.93 , pp. 1787-1789
    • Bunn, H.F.1
  • 9
    • 0019365327 scopus 로고
    • Fetal hemoglobin and clinical severity of homozygous sickle cell disease in early childhood
    • Stevens M.C., Hayes R.J., Vaidya S., Serjeant G.R. Fetal hemoglobin and clinical severity of homozygous sickle cell disease in early childhood. J Pediatr 1981, 98:37-41.
    • (1981) J Pediatr , vol.98 , pp. 37-41
    • Stevens, M.C.1    Hayes, R.J.2    Vaidya, S.3    Serjeant, G.R.4
  • 10
    • 84155186479 scopus 로고    scopus 로고
    • Fetal haemoglobin levels and haematological characteristics of compound heterozygotes for haemoglobin S and deletional hereditary persistence of fetal haemoglobin
    • Ngo D.A., Aygun B., Akinsheye I., et al. Fetal haemoglobin levels and haematological characteristics of compound heterozygotes for haemoglobin S and deletional hereditary persistence of fetal haemoglobin. Br J Haematol 2012, 156:259-264.
    • (2012) Br J Haematol , vol.156 , pp. 259-264
    • Ngo, D.A.1    Aygun, B.2    Akinsheye, I.3
  • 11
    • 0023103593 scopus 로고
    • Subunit assembly of hemoglobin: an important determinant of hematologic phenotype
    • Bunn H.F. Subunit assembly of hemoglobin: an important determinant of hematologic phenotype. Blood 1987, 69:1-6.
    • (1987) Blood , vol.69 , pp. 1-6
    • Bunn, H.F.1
  • 12
    • 3042597832 scopus 로고    scopus 로고
    • A recombinant human hemoglobin with antisickling properties greater than fetal hemoglobin
    • Levasseur D.N., Ryan T.M., Reilly M.P., McCune S.L., Asakura T., Townes T.M. A recombinant human hemoglobin with antisickling properties greater than fetal hemoglobin. J Biol Chem 2004, 279:27518-27524.
    • (2004) J Biol Chem , vol.279 , pp. 27518-27524
    • Levasseur, D.N.1    Ryan, T.M.2    Reilly, M.P.3    McCune, S.L.4    Asakura, T.5    Townes, T.M.6
  • 13
    • 84881262533 scopus 로고    scopus 로고
    • β-globin gene transfer to human bone marrow for sickle cell disease
    • Romero Z., Urbinati F., Geiger S., et al. β-globin gene transfer to human bone marrow for sickle cell disease. J Clin Invest 2013, 123:3317-3330.
    • (2013) J Clin Invest , vol.123 , pp. 3317-3330
    • Romero, Z.1    Urbinati, F.2    Geiger, S.3
  • 14
    • 79953117530 scopus 로고    scopus 로고
    • + cells following lentiviral vector-mediated gene transfer
    • + cells following lentiviral vector-mediated gene transfer. Blood 2011, 117:2817-2826.
    • (2011) Blood , vol.117 , pp. 2817-2826
    • Wilber, A.1    Hargrove, P.W.2    Kim, Y.S.3
  • 15
    • 0037105522 scopus 로고    scopus 로고
    • Development of virus vectors for gene therapy of beta chain hemoglobinopathies: Flanking with a chromatin insulator reduces gamma-globin silencing invivo
    • Emery D.W., Yannaki E., Tubb J., Nishino T., Li Q., Stamatoyannopoulos G. Development of virus vectors for gene therapy of beta chain hemoglobinopathies: Flanking with a chromatin insulator reduces gamma-globin silencing invivo. Blood 2002, 100:2012-2019.
    • (2002) Blood , vol.100 , pp. 2012-2019
    • Emery, D.W.1    Yannaki, E.2    Tubb, J.3    Nishino, T.4    Li, Q.5    Stamatoyannopoulos, G.6
  • 16
    • 58049220057 scopus 로고    scopus 로고
    • Combinatorial incorporation of enhancer-blocking components of the chicken β-globin 5'HS4 and human T-cell receptor α/δ BEAD-1 insulators in self-inactivating retroviral vectors reduces their genotoxic potential
    • Ramezani A., Hawley T.S., Hawley R.G. Combinatorial incorporation of enhancer-blocking components of the chicken β-globin 5'HS4 and human T-cell receptor α/δ BEAD-1 insulators in self-inactivating retroviral vectors reduces their genotoxic potential. Stem Cells 2008, 26:3257-3266.
    • (2008) Stem Cells , vol.26 , pp. 3257-3266
    • Ramezani, A.1    Hawley, T.S.2    Hawley, R.G.3
  • 17
    • 0036200735 scopus 로고    scopus 로고
    • Comparison of various envelope proteins for their ability to pseudotype lentiviral vectors and transducer primitive hematopoietic cells from human blood
    • Hanawa H., Kelly P.F., Nathwani A.C., et al. Comparison of various envelope proteins for their ability to pseudotype lentiviral vectors and transducer primitive hematopoietic cells from human blood. Mol Ther 2002, 5:242-251.
    • (2002) Mol Ther , vol.5 , pp. 242-251
    • Hanawa, H.1    Kelly, P.F.2    Nathwani, A.C.3
  • 18
    • 77951060091 scopus 로고    scopus 로고
    • A zinc-finger transcriptional activator designed to interact with the gamma-globin gene promoters enhances fetal hemoglobin production in primary adult erythroblasts
    • Wilber A., Tschulena U., Hargrove P.W., et al. A zinc-finger transcriptional activator designed to interact with the gamma-globin gene promoters enhances fetal hemoglobin production in primary adult erythroblasts. Blood 2010, 115:3033-3041.
    • (2010) Blood , vol.115 , pp. 3033-3041
    • Wilber, A.1    Tschulena, U.2    Hargrove, P.W.3
  • 19
    • 33750624015 scopus 로고    scopus 로고
    • Cell-culture assays reveal the importance of retroviral vector design for insertional genotoxicity
    • Modlich U., Bohne J., Schmidt M., et al. Cell-culture assays reveal the importance of retroviral vector design for insertional genotoxicity. Blood 2006, 108:2545-2553.
    • (2006) Blood , vol.108 , pp. 2545-2553
    • Modlich, U.1    Bohne, J.2    Schmidt, M.3
  • 20
    • 57849083996 scopus 로고    scopus 로고
    • Human fetal hemoglobin expression is regulated by the developmental stage-specific repressor BCL11A
    • Sankaran V.G., Menne T.F., Xu J., et al. Human fetal hemoglobin expression is regulated by the developmental stage-specific repressor BCL11A. Science 2008, 322:1839-1842.
    • (2008) Science , vol.322 , pp. 1839-1842
    • Sankaran, V.G.1    Menne, T.F.2    Xu, J.3
  • 21
    • 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 2006, 24:89-94.
    • (2006) Nat Biotechnol , vol.24 , pp. 89-94
    • Samakoglu, S.1    Lisowski, L.2    Budak-Alpdogan, T.3


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.