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Blood, 15 December 2003, Vol. 102, No. 13, pp. 4312-4319. Prepublished online as a Blood First Edition Paper on August 21, 2003; DOI 10.1182/blood-2003-04-1251.
GENE THERAPY
Correction of a mouse model of sickle cell disease: lentiviral/antisickling
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  A. Perumbeti, T. Higashimoto, F. Urbinati, R. Franco, H. J. Meiselman, D. Witte, and P. Malik 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, August 6, 2009; 114(6): 1174 - 1185. [Abstract] [Full Text] [PDF]
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H. Zhao, T. I. Pestina, M. Nasimuzzaman, P. Mehta, P. W. Hargrove, and D. A. Persons Amelioration of murine {beta}-thalassemia through drug selection of hematopoietic stem cells transduced with a lentiviral vector encoding both {gamma}-globin and the MGMT drug-resistance gene Blood, June 4, 2009; 113(23): 5747 - 5756. [Abstract] [Full Text] [PDF]
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 Y. R. Su, J. L. Blakemore, Y. Zhang, M. F. Linton, and S. Fazio Lentiviral Transduction of ApoAI Into Hematopoietic Progenitor Cells and Macrophages: Applications to Cell Therapy of Atherosclerosis Arterioscler Thromb Vasc Biol, August 1, 2008; 28(8): 1439 - 1446. [Abstract] [Full Text] [PDF]
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 A. Miccio, R. Cesari, F. Lotti, C. Rossi, F. Sanvito, M. Ponzoni, S. J. E. Routledge, C.-M. Chow, M. N. Antoniou, and G. Ferrari In vivo selection of genetically modified erythroblastic progenitors leads to long-term correction of {beta}-thalassemia PNAS, July 29, 2008; 105(30): 10547 - 10552. [Abstract] [Full Text] [PDF]
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 P. Moi and M. Sadelain Towards the genetic treatment of {beta}-thalassemia: new disease models, new vectors, new cells Haematologica, March 1, 2008; 93(3): 325 - 330. [Full Text] [PDF]
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 T. M. Townes Gene Replacement Therapy for Sickle Cell Disease and Other Blood Disorders Hematology, January 1, 2008; 2008(1): 193 - 196. [Abstract] [Full Text] [PDF]
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L. Lisowski and M. Sadelain Locus control region elements HS1 and HS4 enhance the therapeutic efficacy of globin gene transfer in -thalassemic mice Blood, December 15, 2007; 110(13): 4175 - 4178. [Abstract] [Full Text] [PDF]
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 Y.-W. Pan, J. M. Scarlett, T. T. Luoh, and P. Kurre Prolonged Adherence of Human Immunodeficiency Virus-Derived Vector Particles to Hematopoietic Target Cells Leads to Secondary Transduction In Vitro and In Vivo J. Virol., January 15, 2007; 81(2): 639 - 649. [Abstract] [Full Text] [PDF]
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L.-C. Wu, C.-W. Sun, T. M. Ryan, K. M. Pawlik, J. Ren, and T. M. Townes Correction of sickle cell disease by homologous recombination in embryonic stem cells Blood, August 15, 2006; 108(4): 1183 - 1188. [Abstract] [Full Text] [PDF]
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I. Bot, J. Guo, M. Van Eck, P. J. Van Santbrink, P. H. E. Groot, R. B. Hildebrand, J. Seppen, T. J. C. Van Berkel, and E. A. L. Biessen Lentiviral shRNA silencing of murine bone marrow cell CCR2 leads to persistent knockdown of CCR2 function in vivo Blood, August 15, 2005; 106(4): 1147 - 1153. [Abstract] [Full Text] [PDF]
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M. C. Walters Stem Cell Therapy for Sickle Cell Disease: Transplantation and Gene Therapy Hematology, January 1, 2005; 2005(1): 66 - 73. [Abstract] [Full Text] [PDF]
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G. Puthenveetil, J. Scholes, D. Carbonell, N. Qureshi, P. Xia, L. Zeng, S. Li, Y. Yu, A. L Hiti, J.-K. Yee, et al. Successful correction of the human {beta}-thalassemia major phenotype using a lentiviral vector Blood, December 1, 2004; 104(12): 3445 - 3453. [Abstract] [Full Text] [PDF]
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H. Hanawa, P. W. Hargrove, S. Kepes, D. K. Srivastava, A. W. Nienhuis, and D. A. Persons Extended {beta}-globin locus control region elements promote consistent therapeutic expression of a {gamma}-globin lentiviral vector in murine {beta}-thalassemia Blood, October 15, 2004; 104(8): 2281 - 2290. [Abstract] [Full Text] [PDF]
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 D. N. Levasseur, T. M. Ryan, M. P. Reilly, S. L. McCune, T. Asakura, and T. M. Townes A Recombinant Human Hemoglobin with Anti-sickling Properties Greater than Fetal Hemoglobin J. Biol. Chem., June 25, 2004; 279(26): 27518 - 27524. [Abstract] [Full Text] [PDF]
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H. Hanawa, P. Hematti, K. Keyvanfar, M. E. Metzger, A. Krouse, R. E. Donahue, S. Kepes, J. Gray, C. E. Dunbar, D. A. Persons, et al. Efficient gene transfer into rhesus repopulating hematopoietic stem cells using a simian immunodeficiency virus-based lentiviral vector system Blood, June 1, 2004; 103(11): 4062 - 4069. [Abstract] [Full Text] [PDF]
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| Copyright © 2003 by American Society of Hematology Online ISSN: 1528-0020 | |||||||||
-globin gene transduction of unmobilized, purified hematopoietic stem cells
-globin gene. These cells, which were transduced in the absence of cytokine stimulation, fully reconstitute irradiated recipients and correct the hemolytic anemia and organ pathology that characterize the disease in humans. The mean increase of hemoglobin concentration was 46 g/L (4.6 g/dL) and the average lentiviral copy number was 2.2; therefore, a 21-g/L /vector copy increase (2.1-g/dL) was achieved. This transduction protocol may be directly translatable to patients with sickle cell disease who cannot tolerate current bone marrow mobilization procedures and may not safely be exposed to large viral loads. (Blood. 2003;102:4312-4319)