High-level erythroid lineage-directed gene expression using globin gene regulatory elements after lentiviral vector-mediated gene transfer into primitive human and murine hematopoietic cells

Human Gene Therapy

Volumn 13, Issue 17, 2002, Pages 2007-2016

  Hanawa, Hideki ^a   Persons, Derek A ^a   Nienhuis, Arthur W ^a  

^a ST JUDE CHILDREN S RESEARCH HOSPITAL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ANKYRIN; BETA GLOBIN; GAMMA GLOBIN; GLYCOPHORIN A; GREEN FLUORESCENT PROTEIN; LENTIVIRUS VECTOR; MARKER; PROTEIN SUBUNIT; CD34 ANTIGEN; GLOBIN; GRANULOCYTE COLONY STIMULATING FACTOR; PHOTOPROTEIN;

ANIMAL CELL; ARTICLE; CONTROLLED STUDY; ENHANCER REGION; ERYTHROCYTE; ERYTHROLEUKEMIA CELL; GENE CONTROL; GENE EXPRESSION; GENE INDUCTION; GENE LOCUS; GENE SEQUENCE; GENE TARGETING; GENE TRANSFER; GENETIC ANALYSIS; GENETIC TRANSDUCTION; GENOME; GLOBIN GENE; HEMATOPOIETIC CELL; HUMAN; HUMAN CELL; HUMAN CELL CULTURE; IN VIVO STUDY; INTRON; MOUSE; NONHUMAN; NUCLEOTIDE SEQUENCE; PROMOTER REGION; ANIMAL; BONE MARROW CELL; BONE MARROW TRANSPLANTATION; CELL CULTURE; CELL LINEAGE; DRUG EFFECT; ERYTHROID PRECURSOR CELL; GENE EXPRESSION REGULATION; GENE VECTOR; GENETICS; HEMATOPOIETIC STEM CELL; LENTIVIRINAE; METABOLISM; REGULATOR GENE; VIROLOGY;

ANIMALIA; LENTIVIRUS; MURINAE;

ANIMALS; ANTIGENS, CD34; BONE MARROW CELLS; BONE MARROW TRANSPLANTATION; CELL LINEAGE; ENHANCER ELEMENTS (GENETICS); ERYTHROID PROGENITOR CELLS; GENE EXPRESSION; GENE EXPRESSION REGULATION, VIRAL; GENE TRANSFER TECHNIQUES; GENES, REGULATOR; GENETIC VECTORS; GLOBINS; GRANULOCYTE COLONY-STIMULATING FACTOR; GREEN FLUORESCENT PROTEINS; HEMATOPOIETIC STEM CELLS; HUMANS; INTRONS; LENTIVIRUS; LUMINESCENT PROTEINS; MICE; PROMOTER REGIONS (GENETICS); TRANSDUCTION, GENETIC; TUMOR CELLS, CULTURED;

EID: 0036446597     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/10430340260395866     Document Type: Article

References (35)

1. + cells. J. Virol. 74, 1286-1295.
2. An, D.S., Kung, S.K.P., Bonifacino, A., Wersto, R.P., Metzger, M.E., Agricola, B.A., Mao, S.H., Chen, I.S.Y., and Donahue, R.E. (2001). Lentivirus vector-mediated hematopoietic stem cell gene transfer of common γ-chain cytokine receptor in rhesus macaques. J. Virol. 75, 3547-3555.
3. Antoniou, M., Geraghty, F., Hurst, J., and Grosveld, F. (1998). Efficient 3′-end formation of human beta-globin mRNA in vivo requires sequences within the last intron but occurs independently of the splicing reaction. Nucleic Acids Res. 26, 721-729.
4. Cavazzana-Calvo, M., Hacein-Bey, s., De Saint Basile, G., Gross, F., Yvon, E., Nusbaum, P., Selz, F., Hue, C., Certain, S., Casanova, J.L., Bousso, P., Deist, F.L., and Fischer, A. (2000). Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 288, 669-672.
5. Custodio, N., Carmo-Fonseca, M., Geraghty, F., Pereira, H.S., Grosveld, F., and Antoniou, M. (1999). Inefficient processing impairs release of RNA from the site of transcription. EMBO J. 18, 2855-2866.
6. + cells in the SCID-hu THY/LIV model of human T cell lymphopoiesis. Hum. Gene Ther. 12, 401-413.
7. Emerman, M., and Temin, H.M. (1984). Genes with promoters in retrovirus vectors can be independently suppressed by an epigenetic mechanism. Cell 39, 449-467.
8. + cells transduced by a self-inactivating human immunodeficiency virus type I vector. Hum. Gene Ther. 12, 1079-1089.
9. - bone marrow and cord bloodderived SCID-repopulating cells with third-generation lentiviral vectors. Mol. Ther. 1, 566-573.
10. + cells with H1V-1 derived lentiviral vectors. Mol. Ther. 2, 71-80.
11. Hanawa, H., Kelly, P.F., Nathwani, A.C., Persons, D.A., Carrington, J., Hargrove, P., Vanin, E.F., and Nienhuis, A.W. (2002). Comparison of various envelope proteins for their ability to pseudotype lentiviral vectors and transduce primitive hematopoietic cells from human blood. Mol. Ther. 5, 242-251.
12. Hofmann, W., Schubert, D., Labonte, J., Munson, L., Gibson, S., Scammell, J., Ferrigno, P., and Sodroski, J. (1999). Species-specific, postentry barriers to primate immunodeficiency virus infection. J. Virol. 73, 10020-10028.
13. Holmes, M.L., Haley, J.D., Cerruti, L., Zhou, W.L., Zogos, H., Smith, D.E., Cunningham, J.M., and Jane, S.M. (1999). Identification of Id2 as a globin regulatory protein by representational difference analysis of K562 cells induced to express γ-globin with a fungal compound. Mol. Cell. Biol. 19, 4182-4190.
14. Hu, M., Krause, D., Greaves, M., Sharkis, S., Dexter, M., Heyworth, C., and Enver, T. (1997). Multilineage gene expression precedes commitment in the hemopoietic system. Genes Dev. 11, 774-785.
15. Li, Q., Emery, D.W., Fernandez, M., Han, H., and Stamatoyannopolous, G. (1999). Development of viral vectors for gene therapy of β-chain hemoglobinopathies: Optimization of a γ-globin gene expression cassette. Blood 93, 2208-2216.
16. May, C., Rivella, S., Callegari, J., Heller, G., Gaensler, K.M., Luzzatto, L., and Sadelain, M. (2000). Therapeutic hemoglobin synthesis in β-thalassemic mice expressing lentivirus encoded β-globin. Nature 406, 82-86.
17. Miyoshi, H., Blomer, U., Takahashi, Gage, F.H., and Verma, I.M. (1998). Development of a self-inactivating lentivirus vector. J. Virol. 72, 8150-8157.
18. + cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors. Science 283, 682-686.
19. Moreau-Gaudry, F., Xia, P., Jiang, G., Perelman, N.P., Bauer, G., Ellis, J., Surinya, K.H., Mavilio, F., Shen, C.K., and Malik, P. (2001). High-level erythroid-specific gene expression in primary human and murine hematopoietic cells with selfinactivating lentiviral vectors. Blood 98, 2664-2672.
20. Papayannopoulou, T., Priestly, G.V., Rohde, F., Peterson, K.R., and Nakamoto, B. (2000). Hemopoietic lineage commitment decisions: In vivo evidence from a transgenic mouse model harboring LCR-βpro-LacZ as a transgene. Blood 95, 1274-1282.
21. Pawliuk, R., Westerman, K.A., Fabry, M.E., Payen, E., Tighe, R., Bouhassira, E.E., Acharya, S.A., Ellis, J., London, I.M., Eaves, C.J., Humphries, R.K., Beuzard, Y., Nagel, R.L., and Leboulch, P. (2001). Correction of sickle cell disease in transgenic mouse models by gene therapy. Science 14, 2368-2371.
22. Persons, D.A., Hargrove, P.W., Allay, E.R., Hanawa, H., and Nienhuis, A.W. (2002). The degree of phenotypic correction of murine β-thalassemia following lentiviral-mediated transfer of a human γ-globin genen is influenced by apparent chromosome position effects. Blood. Submitted.
23. Persons, D., Allay, J.A., Riberdy, J.M., Wersto, R.P., Donahue, R.E., Sorrentino, B.P., and Nienhuis, A.W. (1998). Use of the green fluorescent protein as a marker to identify and track genetically modified hematopoietic cells. Nat. Med. 4, 1201-1205.
24. Roe, T., Reynolds, T.C., Yu, G., and Brown, P.O. (1993). Integration of murine leukemia virus DNA depends on mitosis. EMBO J. 12, 2099-2108.
25. Sadelain, M., Wang, C.H., Antoniou, M., Grosveld, F., and Mulligan, R.C. (1995). Generation of a high-titer retroviral vector capable of expressing high levels of the human γ-globin gene. Proc. Natl. Acad. Sci. U.S.A. 92, 6728-6732.
26. Saleque, S., Cameron, S., and Orkin, S.H. (2002). The zinc-finger proto-oncogene Gfi-1b is essential for development of the erythroid and megakaryocytic lineages. Genes Dev. 16, 301-306.
27. Shivdasani, R.A., Rosenblatt, M.F., Zucker-Franklin, D., Jackson, C.W., Hunt, P., Saris, C.J., and Orkin, S.H. (1995). Transcription factor NF-E2 is required for platelet formation independent of the actions of thrombopoietin/MGDF in megakaryocyte development. Cell 81, 695-704.
28. Sorrentino, B.P., and Nienhuis, A.W. (2001). Gene therapy for hematopoietic diseases. In The Molecular Basis of Blood Diseases, 3rd Ed. Stamatoyannopoulos, G., Majerus, P.W., Perlmutter R.M., and Varmus, H., eds. (W. B. Saunders Company, Philadelphia, PA) pp. 969-1003.
29. Takekoshi, K.J., Oh, Y.H., Westerman, K.W., London, I.M., and Leboulch, P. (1995). Retroviral transfer of a human β-globin/δ-globin hybrid gene linked to β locus control region hypersensitive site 2 aimed at the gene therapy of sickle cell disease. Proc. Natl. Acad. Sci. U.S.A. 28, 3014-3018.
30. Tsang, A.P., Fujiwara, Y., Hom, D.B., and Orkin, S.H. (1998). Failure of megakaryopoiesis and arrested erythropoiesis in mice lacking the GATA-1 transcriptional cofactor, FOG. Genes Dev. 12, 1176-1188.
31. 1 human hematopoietic stem cells. Proc. Natl. Acad. Sci. U.S.A. 95, 11939-11944.
32. Vyas, P., McDevitt, M.A., Cantor, A.B., Katz, S.G., Fujiwara, Y., and Orkin, S.H. (1999). Different sequence requirements for expression in erythroid and megakaryocytic cells within a regulatory element upstream of the GATA-1 gene. Development 126, 2799-2881.
33. Woods, N.B., Fahlman, C., Mikkola, H., Hamaguchi, I., Olsson, K., Zufferey, R., Jacobsen, S.E., Trono, D., and Karlsson, S. (2000). Lentiviral gene transfer into primary and secondary NOD/SCID repopulating cells. Blood 96, 3725-3733.
34. Zhou, W., Clouston, D.R., Wang, X., Cerruti. L., Cunningham, J.M., and Jane, S.M. (2000). Induction of human fetal globin gene expression by a novel erythroid factor, NF-E4. Mol. Cell. Biol. 20, 7662-7672.
35. Zufferey, R., Dull, T., Mandel, R.J., Bukovsky, A., Quiroz, D., Naldini, L., and Trono, D. (1998). Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery. J. Virol. 72, 9873-9880.