메뉴 건너뛰기




Volumn 2, Issue , 2015, Pages 15045-

Amelioration of murine sickle cell disease by nonablative conditioning and γ-globin gene-corrected bone marrow cells

Author keywords

[No Author keywords available]

Indexed keywords

CD71 ANTIGEN; HEMOGLOBIN; HEMOGLOBIN F; HEMOGLOBIN GAMMA CHAIN; HEMOSIDERIN; IRON; LENTIVIRUS VECTOR;

EID: 84961657081     PISSN: None     EISSN: 23290501     Source Type: Journal    
DOI: 10.1038/mtm.2015.45     Document Type: Article
Times cited : (16)

References (58)
  • 1
    • 84924418925 scopus 로고    scopus 로고
    • Anemia: progress in molecular mechanisms and therapies
    • 1 Sankaran, VG, Weiss, MJ, Anemia: progress in molecular mechanisms and therapies. Nat Med 21 (2015), 221–230.
    • (2015) Nat Med , vol.21 , pp. 221-230
    • Sankaran, V.G.1    Weiss, M.J.2
  • 2
    • 0030853711 scopus 로고    scopus 로고
    • Pathogenesis and treatment of sickle cell disease
    • 2 Bunn, HF, Pathogenesis and treatment of sickle cell disease. N Engl J Med 337 (1997), 762–769.
    • (1997) N Engl J Med , vol.337 , pp. 762-769
    • Bunn, H.F.1
  • 4
    • 77950622293 scopus 로고    scopus 로고
    • The effect of prolonged administration of hydroxyurea on morbidity and mortality in adult patients with sickle cell syndromes: results of a 17-year, single-center trial (LaSHS)
    • 4 Voskaridou, E, Christoulas, D, Bilalis, A, Plata, E, Varvagiannis, K, Stamatopoulos, G, et al. The effect of prolonged administration of hydroxyurea on morbidity and mortality in adult patients with sickle cell syndromes: results of a 17-year, single-center trial (LaSHS). Blood 115 (2010), 2354–2363.
    • (2010) Blood , vol.115 , pp. 2354-2363
    • Voskaridou, E.1    Christoulas, D.2    Bilalis, A.3    Plata, E.4    Varvagiannis, K.5    Stamatopoulos, G.6
  • 5
    • 0029025475 scopus 로고
    • Effect of hydroxyurea on the frequency of painful crises in sickle cell anemia. Investigators of the Multicenter Study of Hydroxyurea in Sickle Cell Anemia
    • 5 Charache, S, Terrin, ML, Moore, RD, Dover, GJ, Barton, FB, Eckert, SV, et al. Effect of hydroxyurea on the frequency of painful crises in sickle cell anemia. Investigators of the Multicenter Study of Hydroxyurea in Sickle Cell Anemia. N Engl J Med 332 (1995), 1317–1322.
    • (1995) N Engl J Med , vol.332 , pp. 1317-1322
    • Charache, S.1    Terrin, M.L.2    Moore, R.D.3    Dover, G.J.4    Barton, F.B.5    Eckert, S.V.6
  • 6
    • 67651015956 scopus 로고    scopus 로고
    • Fetal hemoglobin chemical inducers for treatment of hemoglobinopathies
    • 6 Testa, U, Fetal hemoglobin chemical inducers for treatment of hemoglobinopathies. Ann Hematol 88 (2009), 505–528.
    • (2009) Ann Hematol , vol.88 , pp. 505-528
    • Testa, U.1
  • 8
    • 0037414164 scopus 로고    scopus 로고
    • Effect of hydroxyurea on mortality and morbidity in adult sickle cell anemia: risks and benefits up to 9 years of treatment
    • 8 Steinberg, MH, Barton, F, Castro, O, Pegelow, CH, Ballas, SK, Kutlar, A, et al. Effect of hydroxyurea on mortality and morbidity in adult sickle cell anemia: risks and benefits up to 9 years of treatment. JAMA 289 (2003), 1645–1651.
    • (2003) JAMA , vol.289 , pp. 1645-1651
    • Steinberg, M.H.1    Barton, F.2    Castro, O.3    Pegelow, C.H.4    Ballas, S.K.5    Kutlar, A.6
  • 9
    • 77953057530 scopus 로고    scopus 로고
    • The risks and benefits of long-term use of hydroxyurea in sickle cell anemia: A 17.5 year follow-up
    • 9 Steinberg, MH, McCarthy, WF, Castro, O, Ballas, SK, Armstrong, FD, Smith, W, et al., Investigators of the Multicenter Study of Hydroxyurea in Sickle Cell Anemia and MSH Patients’ Follow-Up. The risks and benefits of long-term use of hydroxyurea in sickle cell anemia: A 17.5 year follow-up. Am J Hematol 85 (2010), 403–408.
    • (2010) Am J Hematol , vol.85 , pp. 403-408
    • Steinberg, M.H.1    McCarthy, W.F.2    Castro, O.3    Ballas, S.K.4    Armstrong, F.D.5    Smith, W.6
  • 11
    • 38849161460 scopus 로고    scopus 로고
    • Hematopoietic cell transplantation for thalassemia and sickle cell disease: past, present and future
    • 11 Bhatia, M, Walters, MC, Hematopoietic cell transplantation for thalassemia and sickle cell disease: past, present and future. Bone Marrow Transplant 41 (2008), 109–117.
    • (2008) Bone Marrow Transplant , vol.41 , pp. 109-117
    • Bhatia, M.1    Walters, M.C.2
  • 13
    • 84888228928 scopus 로고    scopus 로고
    • Bone marrow transplantation for thalassemia from alternative related donors: improved outcomes with a new approach
    • 13 Gaziev, J, Marziali, M, Isgrò, A, Sodani, P, Paciaroni, K, Gallucci, C, et al. Bone marrow transplantation for thalassemia from alternative related donors: improved outcomes with a new approach. Blood 122 (2013), 2751–2756.
    • (2013) Blood , vol.122 , pp. 2751-2756
    • Gaziev, J.1    Marziali, M.2    Isgrò, A.3    Sodani, P.4    Paciaroni, K.5    Gallucci, C.6
  • 14
    • 72649083282 scopus 로고    scopus 로고
    • Transplantation of unrelated donor umbilical cord blood for nonmalignant diseases: a single institution's experience with 45 patients
    • 14 Jaing, TH, Chen, SH, Tsai, MH, Yang, CP, Hung, IJ, Tsay, PK, Transplantation of unrelated donor umbilical cord blood for nonmalignant diseases: a single institution's experience with 45 patients. Biol Blood Marrow Transplant 16 (2010), 102–107.
    • (2010) Biol Blood Marrow Transplant , vol.16 , pp. 102-107
    • Jaing, T.H.1    Chen, S.H.2    Tsai, M.H.3    Yang, C.P.4    Hung, I.J.5    Tsay, P.K.6
  • 15
    • 0016760198 scopus 로고
    • A new form of hereditary persistence of fetal hemoglobin in blacks and its association with sickle cell trait
    • 15 Stamatoyannopoulos, G, Wood, WG, Papayannopoulou, T, Nute, PE, A new form of hereditary persistence of fetal hemoglobin in blacks and its association with sickle cell trait. Blood 46 (1975), 683–692.
    • (1975) Blood , vol.46 , pp. 683-692
    • Stamatoyannopoulos, G.1    Wood, W.G.2    Papayannopoulou, T.3    Nute, P.E.4
  • 16
    • 0017367181 scopus 로고
    • Heterocellular hereditary persistence of fetal haemoglobin and homozygous sickle-cell disease
    • 16 Serjeant, GR, Serjeant, BE, Mason, K, Heterocellular hereditary persistence of fetal haemoglobin and homozygous sickle-cell disease. Lancet 1 (1977), 795–796.
    • (1977) Lancet , vol.1 , pp. 795-796
    • Serjeant, G.R.1    Serjeant, B.E.2    Mason, K.3
  • 17
    • 0346497365 scopus 로고
    • Common haplotype dependency of high G gamma-globin gene expression and high Hb F levels in beta-thalassemia and sickle cell anemia patients
    • 17 Labie, D, Pagnier, J, Lapoumeroulie, C, Rouabhi, F, Dunda-Belkhodja, O, Chardin, P, et al. Common haplotype dependency of high G gamma-globin gene expression and high Hb F levels in beta-thalassemia and sickle cell anemia patients. Proc Natl Acad Sci USA 82 (1985), 2111–2114.
    • (1985) Proc Natl Acad Sci USA , vol.82 , pp. 2111-2114
    • Labie, D.1    Pagnier, J.2    Lapoumeroulie, C.3    Rouabhi, F.4    Dunda-Belkhodja, O.5    Chardin, P.6
  • 18
    • 0003154471 scopus 로고    scopus 로고
    • The thalassemias
    • Saunders Philadelphia Stamatoyannopoulos G, Majerus P, Perlmutter R, Varmus HE (eds).
    • 18 Weatherall, DJ, The thalassemias. The Molecular Basis for Blood Disorders, 2001, Saunders, Philadelphia, 183–226 Stamatoyannopoulos G, Majerus P, Perlmutter R, Varmus HE (eds).
    • (2001) The Molecular Basis for Blood Disorders , pp. 183-226
    • Weatherall, D.J.1
  • 19
    • 77949846880 scopus 로고    scopus 로고
    • Advances in the understanding of haemoglobin switching
    • 19 Sankaran, VG, Xu, J, Orkin, SH, Advances in the understanding of haemoglobin switching. Br J Haematol 149 (2010), 181–194.
    • (2010) Br J Haematol , vol.149 , pp. 181-194
    • Sankaran, V.G.1    Xu, J.2    Orkin, S.H.3
  • 21
    • 84155186479 scopus 로고    scopus 로고
    • Fetal haemoglobin levels and haematological characteristics of compound heterozygotes for haemoglobin S and deletional hereditary persistence of fetal haemoglobin
    • 21 Ngo, DA, Aygun, B, Akinsheye, I, Hankins, JS, Bhan, I, Luo, HY, et al. Fetal haemoglobin levels and haematological characteristics of compound heterozygotes for haemoglobin S and deletional hereditary persistence of fetal haemoglobin. Br J Haematol 156 (2012), 259–264.
    • (2012) Br J Haematol , vol.156 , pp. 259-264
    • Ngo, D.A.1    Aygun, B.2    Akinsheye, I.3    Hankins, J.S.4    Bhan, I.5    Luo, H.Y.6
  • 22
    • 85176838130 scopus 로고    scopus 로고
    • Gene therapy for severe hemoglobin disorders
    • doi:
    • 22 Villamizar, O, Chambers, CB, Wilber, A, Gene therapy for severe hemoglobin disorders. eLS, 2014 doi: 10.1002/9780470015902.a0025829.
    • (2014) eLS
    • Villamizar, O.1    Chambers, C.B.2    Wilber, A.3
  • 23
    • 0037105522 scopus 로고    scopus 로고
    • Development of virus vectors for gene therapy of beta chain hemoglobinopathies: flanking with a chromatin insulator reduces gamma-globin gene silencing in vivo
    • 23 Emery, DW, 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 gene silencing in vivo. Blood 100 (2002), 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
  • 24
    • 0344305491 scopus 로고    scopus 로고
    • Correction of a mouse model of sickle cell disease: lentiviral/antisickling beta-globin gene transduction of unmobilized, purified hematopoietic stem cells
    • 24 Levasseur, DN, Ryan, TM, Pawlik, KM, Townes, TM, Correction of a mouse model of sickle cell disease: lentiviral/antisickling beta-globin gene transduction of unmobilized, purified hematopoietic stem cells. Blood 102 (2003), 4312–4319.
    • (2003) Blood , vol.102 , pp. 4312-4319
    • Levasseur, D.N.1    Ryan, T.M.2    Pawlik, K.M.3    Townes, T.M.4
  • 25
    • 0035861452 scopus 로고    scopus 로고
    • Correction of sickle cell disease in transgenic mouse models by gene therapy
    • 25 Pawliuk, R, Westerman, KA, Fabry, ME, Payen, E, Tighe, R, Bouhassira, EE, et al. Correction of sickle cell disease in transgenic mouse models by gene therapy. Science 294 (2001), 2368–2371.
    • (2001) Science , vol.294 , pp. 2368-2371
    • Pawliuk, R.1    Westerman, K.A.2    Fabry, M.E.3    Payen, E.4    Tighe, R.5    Bouhassira, E.E.6
  • 26
    • 63649132974 scopus 로고    scopus 로고
    • Correction of murine sickle cell disease using gamma-globin lentiviral vectors to mediate high-level expression of fetal hemoglobin
    • 26 Pestina, TI, Hargrove, PW, Jay, D, Gray, JT, Boyd, KM, Persons, DA, Correction of murine sickle cell disease using gamma-globin lentiviral vectors to mediate high-level expression of fetal hemoglobin. Mol Ther 17 (2009), 245–252.
    • (2009) Mol Ther , vol.17 , pp. 245-252
    • Pestina, T.I.1    Hargrove, P.W.2    Jay, D.3    Gray, J.T.4    Boyd, K.M.5    Persons, D.A.6
  • 27
    • 69849094491 scopus 로고    scopus 로고
    • A novel human gamma-globin gene vector for genetic correction of sickle cell anemia in a humanized sickle mouse model: critical determinants for successful correction
    • 27 Perumbeti, A, Higashimoto, T, Urbinati, F, Franco, R, Meiselman, HJ, Witte, D, et al. A novel human gamma-globin gene vector for genetic correction of sickle cell anemia in a humanized sickle mouse model: critical determinants for successful correction. Blood 114 (2009), 1174–1185.
    • (2009) Blood , vol.114 , pp. 1174-1185
    • Perumbeti, A.1    Higashimoto, T.2    Urbinati, F.3    Franco, R.4    Meiselman, H.J.5    Witte, D.6
  • 28
    • 80051562984 scopus 로고    scopus 로고
    • Allogeneic hematopoietic stem cell transplantation for sickle cell disease: the time is now
    • 28 Hsieh, MM, Fitzhugh, CD, Tisdale, JF, Allogeneic hematopoietic stem cell transplantation for sickle cell disease: the time is now. Blood 118 (2011), 1197–1207.
    • (2011) Blood , vol.118 , pp. 1197-1207
    • Hsieh, M.M.1    Fitzhugh, C.D.2    Tisdale, J.F.3
  • 30
    • 84903625438 scopus 로고    scopus 로고
    • Nonmyeloablative HLA-matched sibling allogeneic hematopoietic stem cell transplantation for severe sickle cell phenotype
    • 30 Hsieh, MM, Fitzhugh, CD, Weitzel, RP, Link, ME, Coles, WA, Zhao, X, et al. Nonmyeloablative HLA-matched sibling allogeneic hematopoietic stem cell transplantation for severe sickle cell phenotype. JAMA 312 (2014), 48–56.
    • (2014) JAMA , vol.312 , pp. 48-56
    • Hsieh, M.M.1    Fitzhugh, C.D.2    Weitzel, R.P.3    Link, M.E.4    Coles, W.A.5    Zhao, X.6
  • 31
    • 84929047585 scopus 로고    scopus 로고
    • Potentially therapeutic levels of anti-sickling globin gene expression following lentivirus-mediated gene transfer in sickle cell disease bone marrow CD34+ cells
    • 31 Urbinati, F, Hargrove, PW, Geiger, S, Romero, Z, Wherley, J, Kaufman, ML, et al. Potentially therapeutic levels of anti-sickling globin gene expression following lentivirus-mediated gene transfer in sickle cell disease bone marrow CD34+ cells. Exp Hematol 43 (2015), 346–351.
    • (2015) Exp Hematol , vol.43 , pp. 346-351
    • Urbinati, F.1    Hargrove, P.W.2    Geiger, S.3    Romero, Z.4    Wherley, J.5    Kaufman, M.L.6
  • 32
    • 79953117530 scopus 로고    scopus 로고
    • Therapeutic levels of fetal hemoglobin in erythroid progeny of β-thalassemic CD34+ cells after lentiviral vector-mediated gene transfer
    • 32 Wilber, A, Hargrove, PW, Kim, YS, Riberdy, JM, Sankaran, VG, Papanikolaou, E, et al. Therapeutic levels of fetal hemoglobin in erythroid progeny of β-thalassemic CD34+ cells after lentiviral vector-mediated gene transfer. Blood 117 (2011), 2817–2826.
    • (2011) Blood , vol.117 , pp. 2817-2826
    • Wilber, A.1    Hargrove, P.W.2    Kim, Y.S.3    Riberdy, J.M.4    Sankaran, V.G.5    Papanikolaou, E.6
  • 33
    • 33846909503 scopus 로고    scopus 로고
    • A role for mammalian target of rapamycin in regulating T cell activation versus anergy
    • 33 Zheng, Y, Collins, SL, Lutz, MA, Allen, AN, Kole, TP, Zarek, PE, et al. A role for mammalian target of rapamycin in regulating T cell activation versus anergy. J Immunol 178 (2007), 2163–2170.
    • (2007) J Immunol , vol.178 , pp. 2163-2170
    • Zheng, Y.1    Collins, S.L.2    Lutz, M.A.3    Allen, A.N.4    Kole, T.P.5    Zarek, P.E.6
  • 34
    • 84878359026 scopus 로고    scopus 로고
    • Splenic complications of sickle cell anemia and the role of splenectomy
    • 34 Al-Salem, AH, Splenic complications of sickle cell anemia and the role of splenectomy. ISRN Hematol, 2011, 2011, 864257.
    • (2011) ISRN Hematol , vol.2011 , pp. 864257
    • Al-Salem, A.H.1
  • 35
    • 59049085233 scopus 로고    scopus 로고
    • Sickle cell disease and the kidney
    • 35 Scheinman, JI, Sickle cell disease and the kidney. Nat Clin Pract Nephrol 5 (2009), 78–88.
    • (2009) Nat Clin Pract Nephrol , vol.5 , pp. 78-88
    • Scheinman, J.I.1
  • 37
    • 84911973714 scopus 로고    scopus 로고
    • Predictors of splenic function preservation in children with sickle cell anemia treated with hydroxyurea
    • 37 Nottage, KA, Ware, RE, Winter, B, Smeltzer, M, Wang, WC, Hankins, JS, et al. Predictors of splenic function preservation in children with sickle cell anemia treated with hydroxyurea. Eur J Haematol 93 (2014), 377–383.
    • (2014) Eur J Haematol , vol.93 , pp. 377-383
    • Nottage, K.A.1    Ware, R.E.2    Winter, B.3    Smeltzer, M.4    Wang, W.C.5    Hankins, J.S.6
  • 40
    • 0031870487 scopus 로고    scopus 로고
    • Haematopoietic stem cell transplantation for sickle cell anaemia: the first 50 patients transplanted in Belgium
    • 40 Vermylen, C, Cornu, G, Ferster, A, Brichard, B, Ninane, J, Ferrant, A, et al. Haematopoietic stem cell transplantation for sickle cell anaemia: the first 50 patients transplanted in Belgium. Bone Marrow Transplant 22 (1998), 1–6.
    • (1998) Bone Marrow Transplant , vol.22 , pp. 1-6
    • Vermylen, C.1    Cornu, G.2    Ferster, A.3    Brichard, B.4    Ninane, J.5    Ferrant, A.6
  • 41
    • 34948830175 scopus 로고    scopus 로고
    • Long-term results of related myeloablative stem-cell transplantation to cure sickle cell disease
    • 41 Bernaudin, F, Socie, G, Kuentz, M, Chevret, S, Duval, M, Bertrand, Y, et al., SFGM-TC. Long-term results of related myeloablative stem-cell transplantation to cure sickle cell disease. Blood 110 (2007), 2749–2756.
    • (2007) Blood , vol.110 , pp. 2749-2756
    • Bernaudin, F.1    Socie, G.2    Kuentz, M.3    Chevret, S.4    Duval, M.5    Bertrand, Y.6
  • 43
    • 33845725212 scopus 로고    scopus 로고
    • Sirolimus promotes tolerance for donor and recipient antigens after MHC class II disparate bone marrow transplantation in rats
    • 43 Jäger, MD, Liu, JY, Timrott, KF, Popp, FC, Stoeltzing, O, Lang, SA, et al. Sirolimus promotes tolerance for donor and recipient antigens after MHC class II disparate bone marrow transplantation in rats. Exp Hematol 35 (2007), 164–170.
    • (2007) Exp Hematol , vol.35 , pp. 164-170
    • Jäger, M.D.1    Liu, J.Y.2    Timrott, K.F.3    Popp, F.C.4    Stoeltzing, O.5    Lang, S.A.6
  • 44
    • 33846217342 scopus 로고    scopus 로고
    • Induction of chimerism in rhesus macaques through stem cell transplant and costimulation blockade-based immunosuppression
    • 44 Kean, LS, Adams, AB, Strobert, E, Hendrix, R, Gangappa, S, Jones, TR, et al. Induction of chimerism in rhesus macaques through stem cell transplant and costimulation blockade-based immunosuppression. Am J Transplant 7 (2007), 320–335.
    • (2007) Am J Transplant , vol.7 , pp. 320-335
    • Kean, L.S.1    Adams, A.B.2    Strobert, E.3    Hendrix, R.4    Gangappa, S.5    Jones, T.R.6
  • 45
    • 77956928344 scopus 로고    scopus 로고
    • Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia
    • 45 Cavazzana-Calvo, M, Payen, E, Negre, O, Wang, G, Hehir, K, Fusil, F, et al. Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia. Nature 467 (2010), 318–322.
    • (2010) Nature , vol.467 , pp. 318-322
    • Cavazzana-Calvo, M.1    Payen, E.2    Negre, O.3    Wang, G.4    Hehir, K.5    Fusil, F.6
  • 46
    • 84878668046 scopus 로고    scopus 로고
    • Advances in stem cell transplantation and gene therapy in the β-hemoglobinopathies
    • 46 Payen, E, Leboulch, P, Advances in stem cell transplantation and gene therapy in the β-hemoglobinopathies. Hematology Am Soc Hematol Educ Program 2012 (2012), 276–283.
    • (2012) Hematology Am Soc Hematol Educ Program , vol.2012 , pp. 276-283
    • Payen, E.1    Leboulch, P.2
  • 47
    • 84926339367 scopus 로고    scopus 로고
    • Preclinical evaluation of efficacy and safety of an improved lentiviral vector for the treatment of β-thalassemia and sickle cell disease
    • 47 Negre, O, Bartholomae, C, Beuzard, Y, Cavazzana, M, Christiansen, L, Courne, C, et al. Preclinical evaluation of efficacy and safety of an improved lentiviral vector for the treatment of β-thalassemia and sickle cell disease. Curr Gene Ther 15 (2015), 64–81.
    • (2015) Curr Gene Ther , vol.15 , pp. 64-81
    • Negre, O.1    Bartholomae, C.2    Beuzard, Y.3    Cavazzana, M.4    Christiansen, L.5    Courne, C.6
  • 48
    • 84964762224 scopus 로고    scopus 로고
    • Initial results from the Northstar Study (HGB-204): a phase 1/2 study of gene therapy for β-thalassemia major via transplantation of autologous hematopoietic stem cells transduced ex vivo with a lentiviral βA-T87Q-globin vector (Lentiglobin BB305 drug product) [abstract]
    • 48 Thompson, AA, Rasko, JE, Hongeng, S, et al. Initial results from the Northstar Study (HGB-204): a phase 1/2 study of gene therapy for β-thalassemia major via transplantation of autologous hematopoietic stem cells transduced ex vivo with a lentiviral βA-T87Q-globin vector (Lentiglobin BB305 drug product) [abstract]. Blood, 124, 2014, 549.
    • (2014) Blood , vol.124 , pp. 549
    • Thompson, A.A.1    Rasko, J.E.2    Hongeng, S.3
  • 49
    • 0021343093 scopus 로고
    • Is there a threshold level of fetal hemoglobin that ameliorates morbidity in sickle cell anemia?
    • 49 Powars, DR, Weiss, JN, Chan, LS, Schroeder, WA, Is there a threshold level of fetal hemoglobin that ameliorates morbidity in sickle cell anemia?. Blood 63 (1984), 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
  • 50
    • 0033560094 scopus 로고    scopus 로고
    • Induction of fetal hemoglobin in sickle cell disease
    • 50 Bunn, HF, Induction of fetal hemoglobin in sickle cell disease. Blood 93 (1999), 1787–1789.
    • (1999) Blood , vol.93 , pp. 1787-1789
    • Bunn, H.F.1
  • 51
    • 0019365327 scopus 로고
    • Fetal hemoglobin and clinical severity of homozygous sickle cell disease in early childhood
    • 51 Stevens, MC, Hayes, RJ, Vaidya, S, Serjeant, GR, Fetal hemoglobin and clinical severity of homozygous sickle cell disease in early childhood. J Pediatr 98 (1981), 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
  • 52
    • 0027233846 scopus 로고
    • Sparing effect of hemoglobin F and hemoglobin A2 on the polymerization of hemoglobin S at physiologic ligand saturations
    • 52 Poillon, WN, Kim, BC, Rodgers, GP, Noguchi, CT, Schechter, AN, Sparing effect of hemoglobin F and hemoglobin A2 on the polymerization of hemoglobin S at physiologic ligand saturations. Proc Natl Acad Sci USA 90 (1993), 5039–5043.
    • (1993) Proc Natl Acad Sci USA , vol.90 , pp. 5039-5043
    • Poillon, W.N.1    Kim, B.C.2    Rodgers, G.P.3    Noguchi, C.T.4    Schechter, A.N.5
  • 54
    • 85176855765 scopus 로고    scopus 로고
    • Clinical, Accessed 6 March Trials.gov
    • 54 National Institutes of Health. Clinical Trials.gov https://clinicaltrials.gov/ct2/results?term=sickle+cell+anemia+AND+gene+therapy&recr=Open, Accessed 6 March 2015.
    • (2015)
  • 55
    • 84928554563 scopus 로고    scopus 로고
    • Enrichment of human hematopoietic stem/progenitor cells facilitates transduction for stem cell gene therapy
    • 55 Baldwin, K, Urbinati, F, Romero, Z, Campo-Fernandez, B, Kaufman, ML, Cooper, AR, et al. Enrichment of human hematopoietic stem/progenitor cells facilitates transduction for stem cell gene therapy. Stem Cells 33 (2015), 1532–1542.
    • (2015) Stem Cells , vol.33 , pp. 1532-1542
    • Baldwin, K.1    Urbinati, F.2    Romero, Z.3    Campo-Fernandez, B.4    Kaufman, M.L.5    Cooper, A.R.6
  • 56
    • 84928470127 scopus 로고    scopus 로고
    • Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells
    • 56 Hoban, MD, Cost, GJ, Mendel, MC, Romero, Z, Kaufman, ML, Joglekar, AV, et al. Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells. Blood 125 (2015), 2597–2604.
    • (2015) Blood , vol.125 , pp. 2597-2604
    • Hoban, M.D.1    Cost, G.J.2    Mendel, M.C.3    Romero, Z.4    Kaufman, M.L.5    Joglekar, A.V.6
  • 57
    • 0036200735 scopus 로고    scopus 로고
    • Comparison of various envelope proteins for their ability to pseudotype lentiviral vectors and transduce primitive hematopoietic cells from human blood
    • 57 Hanawa, H, Kelly, PF, Nathwani, AC, Persons, DA, Vandergriff, JA, Hargrove, P, et al. Comparison of various envelope proteins for their ability to pseudotype lentiviral vectors and transduce primitive hematopoietic cells from human blood. Mol Ther 5 (2002), 242–251.
    • (2002) Mol Ther , vol.5 , pp. 242-251
    • Hanawa, H.1    Kelly, P.F.2    Nathwani, A.C.3    Persons, D.A.4    Vandergriff, J.A.5    Hargrove, P.6
  • 58
    • 0037772187 scopus 로고    scopus 로고
    • Successful treatment of murine beta-thalassemia using in vivo selection of genetically modified, drug-resistant hematopoietic stem cells
    • 58 Persons, DA, Allay, ER, Sawai, N, Hargrove, PW, Brent, TP, Hanawa, H, et al. Successful treatment of murine beta-thalassemia using in vivo selection of genetically modified, drug-resistant hematopoietic stem cells. Blood 102 (2003), 506–513.
    • (2003) Blood , vol.102 , pp. 506-513
    • Persons, D.A.1    Allay, E.R.2    Sawai, N.3    Hargrove, P.W.4    Brent, T.P.5    Hanawa, H.6


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.