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Journal Abstract Search


301 related items for PubMed ID: 19074080

  • 1. Gene replacement therapy for sickle cell disease and other blood disorders.
    Townes TM.
    Hematology Am Soc Hematol Educ Program; 2008; ():193-6. PubMed ID: 19074080
    [Abstract] [Full Text] [Related]

  • 2. Correction of sickle cell disease by homologous recombination in embryonic stem cells.
    Wu LC, Sun CW, Ryan TM, Pawlik KM, Ren J, Townes TM.
    Blood; 2006 Aug 15; 108(4):1183-8. PubMed ID: 16638928
    [Abstract] [Full Text] [Related]

  • 3. Gene Therapy for Sickle Cell Disease: A Lentiviral Vector Comparison Study.
    Urbinati F, Campo Fernandez B, Masiuk KE, Poletti V, Hollis RP, Koziol C, Kaufman ML, Brown D, Mavilio F, Kohn DB.
    Hum Gene Ther; 2018 Oct 15; 29(10):1153-1166. PubMed ID: 30198339
    [Abstract] [Full Text] [Related]

  • 4. Correction of sickle cell disease in transgenic mouse models by gene therapy.
    Pawliuk R, Westerman KA, Fabry ME, Payen E, Tighe R, Bouhassira EE, Acharya SA, Ellis J, London IM, Eaves CJ, Humphries RK, Beuzard Y, Nagel RL, Leboulch P.
    Science; 2001 Dec 14; 294(5550):2368-71. PubMed ID: 11743206
    [Abstract] [Full Text] [Related]

  • 5. Correction of a mouse model of sickle cell disease: lentiviral/antisickling beta-globin gene transduction of unmobilized, purified hematopoietic stem cells.
    Levasseur DN, Ryan TM, Pawlik KM, Townes TM.
    Blood; 2003 Dec 15; 102(13):4312-9. PubMed ID: 12933581
    [Abstract] [Full Text] [Related]

  • 6. Genetic strategies for the treatment of sickle cell anaemia.
    Mansilla-Soto J, Rivière I, Sadelain M.
    Br J Haematol; 2011 Sep 15; 154(6):715-27. PubMed ID: 21707580
    [Abstract] [Full Text] [Related]

  • 7. Potentially therapeutic levels of anti-sickling globin gene expression following lentivirus-mediated gene transfer in sickle cell disease bone marrow CD34+ cells.
    Urbinati F, Hargrove PW, Geiger S, Romero Z, Wherley J, Kaufman ML, Hollis RP, Chambers CB, Persons DA, Kohn DB, Wilber A.
    Exp Hematol; 2015 May 15; 43(5):346-351. PubMed ID: 25681747
    [Abstract] [Full Text] [Related]

  • 8. Correction of the sickle cell mutation in embryonic stem cells.
    Chang JC, Ye L, Kan YW.
    Proc Natl Acad Sci U S A; 2006 Jan 24; 103(4):1036-40. PubMed ID: 16407095
    [Abstract] [Full Text] [Related]

  • 9. Stem cell therapy for sickle cell disease: transplantation and gene therapy.
    Walters MC.
    Hematology Am Soc Hematol Educ Program; 2005 Jan 24; ():66-73. PubMed ID: 16304361
    [Abstract] [Full Text] [Related]

  • 10. Correction of murine sickle cell disease using gamma-globin lentiviral vectors to mediate high-level expression of fetal hemoglobin.
    Pestina TI, Hargrove PW, Jay D, Gray JT, Boyd KM, Persons DA.
    Mol Ther; 2009 Feb 24; 17(2):245-52. PubMed ID: 19050697
    [Abstract] [Full Text] [Related]

  • 11. High-level beta-globin expression and preferred intragenic integration after lentiviral transduction of human cord blood stem cells.
    Imren S, Fabry ME, Westerman KA, Pawliuk R, Tang P, Rosten PM, Nagel RL, Leboulch P, Eaves CJ, Humphries RK.
    J Clin Invest; 2004 Oct 24; 114(7):953-62. PubMed ID: 15467834
    [Abstract] [Full Text] [Related]

  • 12. Hematopoietic stem cell gene transfer for the treatment of hemoglobin disorders.
    Persons DA.
    Hematology Am Soc Hematol Educ Program; 2009 Oct 24; ():690-7. PubMed ID: 20008255
    [Abstract] [Full Text] [Related]

  • 13. Therapeutic hemoglobin levels after gene transfer in β-thalassemia mice and in hematopoietic cells of β-thalassemia and sickle cells disease patients.
    Breda L, Casu C, Gardenghi S, Bianchi N, Cartegni L, Narla M, Yazdanbakhsh K, Musso M, Manwani D, Little J, Gardner LB, Kleinert DA, Prus E, Fibach E, Grady RW, Giardina PJ, Gambari R, Rivella S.
    PLoS One; 2012 Oct 24; 7(3):e32345. PubMed ID: 22479321
    [Abstract] [Full Text] [Related]

  • 14. Highly efficient editing of the β-globin gene in patient-derived hematopoietic stem and progenitor cells to treat sickle cell disease.
    Park SH, Lee CM, Dever DP, Davis TH, Camarena J, Srifa W, Zhang Y, Paikari A, Chang AK, Porteus MH, Sheehan VA, Bao G.
    Nucleic Acids Res; 2019 Sep 05; 47(15):7955-7972. PubMed ID: 31147717
    [Abstract] [Full Text] [Related]

  • 15. Improved Titer and Gene Transfer by Lentiviral Vectors Using Novel, Small β-Globin Locus Control Region Elements.
    Morgan RA, Unti MJ, Aleshe B, Brown D, Osborne KS, Koziol C, Ayoub PG, Smith OB, O'Brien R, Tam C, Miyahira E, Ruiz M, Quintos JP, Senadheera S, Hollis RP, Kohn DB.
    Mol Ther; 2020 Jan 08; 28(1):328-340. PubMed ID: 31628051
    [Abstract] [Full Text] [Related]

  • 16. Expression of an anti-sickling beta-globin in human erythroblasts derived from retrovirally transduced primitive normal and sickle cell disease hematopoietic cells.
    Oh IH, Fabry ME, Humphries RK, Pawliuk R, Leboulch P, Hoffman R, Nagel RL, Eaves C.
    Exp Hematol; 2004 May 08; 32(5):461-9. PubMed ID: 15145214
    [Abstract] [Full Text] [Related]

  • 17. Gene Therapy for beta-thalassemia.
    Malik P, Arumugam PI.
    Hematology Am Soc Hematol Educ Program; 2005 May 08; ():45-50. PubMed ID: 16304358
    [Abstract] [Full Text] [Related]

  • 18. A phase I/II clinical trial of beta-globin gene therapy for beta-thalassemia.
    Bank A, Dorazio R, Leboulch P.
    Ann N Y Acad Sci; 2005 May 08; 1054():308-16. PubMed ID: 16339679
    [Abstract] [Full Text] [Related]

  • 19. Successful correction of the human Cooley's anemia beta-thalassemia major phenotype using a lentiviral vector flanked by the chicken hypersensitive site 4 chromatin insulator.
    Malik P, Arumugam PI, Yee JK, Puthenveetil G.
    Ann N Y Acad Sci; 2005 May 08; 1054():238-49. PubMed ID: 16339671
    [Abstract] [Full Text] [Related]

  • 20. 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.
    Perumbeti A, Higashimoto T, Urbinati F, Franco R, Meiselman HJ, Witte D, Malik P.
    Blood; 2009 Aug 06; 114(6):1174-85. PubMed ID: 19474450
    [Abstract] [Full Text] [Related]


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