Adeno-associated virus 2-mediated transduction and erythroid cell-specific expression of a human β-globin gene

Gene Therapy

Volumn 3, Issue 3, 1996, Pages 223-229

  Zhou, S Z ^a   Li, Q ^b   Stamatoyannopoulos, G ^b   Srivastava, A ^a,c  

^a INDIANA UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b UNIVERSITY OF WASHINGTON SCHOOL OF MEDICINE   (United States)

^c INDIANA UNIVERSITY   (United States)

Author keywords

globin; AAV; Gene therapy; Hemoglobinopathies; Viral vectors

Indexed keywords

BETA GLOBIN; NEOMYCIN; THYMIDINE KINASE;

ADENO ASSOCIATED VIRUS; ANTIBIOTIC RESISTANCE; ARTICLE; BACTERIAL GENE; CANCER CELL CULTURE; CONTROLLED STUDY; DNA INTEGRATION; DNA PROBE; ERYTHROID CELL; ERYTHROLEUKEMIA CELL; EXPRESSION VECTOR; GENE EXPRESSION; GENETIC TRANSDUCTION; GLOBIN GENE; HERPES VIRUS; HUMAN; HUMAN CELL; NASOPHARYNX CARCINOMA; NONHUMAN; NORTHERN BLOTTING; PRIORITY JOURNAL; PROMOTER REGION; SOUTHERN BLOTTING; VIRUS RECOMBINANT;

DEPENDOVIRUS; GLOBINS; HUMANS; LEUKEMIA, ERYTHROBLASTIC, ACUTE; RECOMBINATION, GENETIC; TRANSDUCTION, GENETIC; TUMOR CELLS, CULTURED;

EID: 0029944720     PISSN: 09697128     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Article

References (54)

1. Stamatoyannopoulos G, Nienhuis AW. In: Stamatoyannopoulos G, Nienhuis AW, Maserus PW, Varmus HE (eds). The molecular basis of blood diseases. WB Saunders Co: Philadelphia, 1994, pp 107-155.
2. Walsh CE, Miller JL, Nienhuis AW, Samulski RJ. Gene therapy for human hemoglobinopathies. Proc Soc Exp Biol Med 1993; 204: 289-300.
3. Cone RD, Weber-Benarous A, Baorto D, Mulligan RC. Regulated expression of a complete human β-globin gene encoded by a transmissible retrovirus vector. Mol Cell Biol 1987; 7: 887-897.
4. Karlsson S et al. Retroviral-mediated transfer of genomic globin genes leads to regulated production of RNA and protein. Proc Natl Acad Sci USA 1987; 84: 2411-2415.
5. Miller AD et al. Design of retrovirus vector for transfer and expression of the human β-globin gene. J Virol 1988; 62: 4337-4345.
6. Bender MA, Miller AD, Gelinas RE. Expression of the human β-globin gene after retroviral transfer into murine erythroleukemia cells and human BFU-E cells. Mol Cell Biol 1988; 8: 1725-1735.
7. Dzierzak EA, Papayannopoulou T, Mulligan RC. Lineage-specific expression of a human β-globin gene in murine bone marrow transplant recipients reconstituted with retrovirus-transduced stem cells. Nature 1988; 331: 35-41.
8. Karlsson S et al. Expression of the human β-globin gene following retroviral-mediated multipotential hematopoietic progenitors of mice. Proc Natl Acad Sci USA 1988; 85: 6062-6066.
9. Bender MA, Gelinas RE, Miller AD. A majority of mice show long-term expression of a human β-globin gene into hematopoietic stem cells. Mol Cell Biol 1989; 9: 1426-1434.
10. Tuan DYH, Solomon WB, London IM, Lee DP. An erythroid specific, development stage-independent enhancer far upstream of the human 'β-like globin' genes. Proc Natl Acad Sci USA 1989; 86: 2554-2559.
11. Novak U et al. High-level β-globin expression after retroviral transfer of locus activation region-containing human β-globin gene derivatives into murine erythroleukemia cells. Proc Natl Acad Sci USA 1990; 87: 3386-3390.
12. Chang JC, Liu D, Kan YW. A 36 base pair core sequence of locus control region enhances retrovirally transferred human β-globin gene expression. Proc Natl Acad Sci USA 1992; 89: 3107-3110
13. Plavec I, Papayannopoulou T, Maury C, Meyer F. A human β-globin gene fused to the human β-globin locus control region is expressed at high levels in erythroid cells of mice engrafted with retrovirus transduced hematopoietic stem cells. Blood 1993, 81: 1384-1392.
14. Leboulch P et al. Mutagenesis of retroviral vectors transducing human β-globin gene and β-globin locus control region derivatives results in stable transmission of an active transcriptional structure. EMBO J 1994; 13: 3065-3076.
15. Blacklow NR et al. A sero-epidemiologic study of adeno-associated virus infection in infants and children. Am J Epidemiol 1971; 94: 359-366.
16. Brown KE, Young NS, Liu JM. Molecular, cellular and clinical aspects of parvovirus B19 infection. Crit Rev Oncol Hematol 1994; 16: 1-31.
17. Ozawa K, Kurtzman GJ, Young NS. Productive infection by B19 parvovirus of human erythroid bone marrow cells in vitro Blood 1987; 70: 384-391.
18. Srivastava A, Lu L. Replication of B19 parvovirus in highly enriched hematopoietic progenitor cells from normal human bone marrow. J Virol 1988; 62: 3059-3063.
19. Yaegashi N et al. Propagation of human parvovirus B19 in primary culture of erythroid lineage cells derived from fetal liver. J Virol 1989; 63: 2422-2426.
20. Takahashi T et al. Susceptibility of human erythropoietic cells to B19 parvovirus in vitro increases with differentiation. Blood 1990; 5: 603-610.
21. Srivastava CH, Zhou SZ, Munshi NC, Srivastava A. Parvovirus B19 replication in human umbilical cord blood cells. Virology 1992; 189: 456-461.
22. Srivastava CH et al. Construction of a recombinant human parvovirus B19: adeno-associated virus 2 (AAV) DNA inverted terminal repeats are functional in an AAV-B19 hybrid virus. Proc Natl Acad Sci USA 1989; 86: 8078-8082.
23. Berns KI and Bohenzky RA. Adeno-associated viruses: an update. Adv Virus Res 1987; 32: 243-306.
24. Kotin RM et al. Site-specific integration by adeno-associated virus. Proc Natl Acad Sci USA 1990; 87: 2211-2215.
25. Kotin RM, Menninger JC, Ward DC, Berns KI. Mapping and direct visualization of a region-specific viral DNA integration site on chromosome 19q13-qter. Genomics 1991; 10: 831-834.
26. Samulski RJ et al. Targeted integration of adeno-associated virus (AAV) into human chromosome 19. EMBO J 1991; 10: 3941-3950.
27. Muro-Cacho CA, Samulski RJ, Kaplan D. Gene transfer in human lymphocytes using a vector based on adeno-associated virus. J Immunother 1992; 11: 231-235.
28. Muzyczka N. Use of adeno-associated virus as a general transduction vector for mammalian cells Curr Top Microbiol Immunol 1992; 158: 98-129.
29. Carter BJ. Adeno-associated virus vectors. Curr Opin Biotechnol 1993; 3: 533-538.
30. Srivastava A. Parvovirus-based vectors for human gene therapy. Blood Cells 1994; 20: 531-538.
31. Charnay P, Maniatis T. Transcriptional regulation of globin gene expression in the human erythroid cell line K562. Science 1983; 220: 1281-1283.
32. Zhou SZ et al. Adeno-associated virus 2-mediated high efficiency gene transfer into immature and mature subsets of hematopoietic progenitor cells in human umbilical cord blood. J Exp Med 1994; 179: 1867-1875.
33. Ponnazhagan S et al. Differential expression from parvovirus B19p6 promoter in human cells following plasmid transfection and recombinant adeno-associated virus 2 (AAV) infection: human megakaryocytic leukemia cells are non-permissive for AAV infection. J Gen Virol (submitted).
34. Walsh CE et al. Regulated high level expression of a human γ-globin gene introduced into erythroid cells by an adeno-associated virus vector. Proc Natl Acad Sci USA 1992; 89: 7257-7261.
35. Miller JL et al. Recombinant adeno-associated virus (rAAV)-mediated expression of a human γ-globin gene in human progenitor-derived erythroid cells. Proc Natl Acad Sci USA 1994; 91: 10183-10187.
36. Dixit M, Webb MS, Smart WC, Ohi S. Construction and expression of a recombinanf adeno-associated vims that harbors a human β-globin-encoding cDNA. Gene 1991; 104: 253-257.
37. Einerhand MPW et al. Regulated high-level human β-globin gene expression in erythroid cells following recombinant adenoassociated virus-mediated gene transfer. Gene Therapy 1995; 2: 336-343.
38. Ponnazhagan S et al. Lack of site-specific integration of the recombinant adeno-associated virus 2 genomes in human cells. Hum Gene Ther 1995 (submitted).
39. Goodman S et al. Recombinant adeno-associated virus mediated gene transfer into hematopoietic progenitor cells. Blood 1994; 84: 1492-1500.
40. Russell DW, Miller AD, Alexander IE. Adeno-associated virus vectors preferentially transduce cells in S phase. Proc Natl Acad Sci USA 1994; 91: 8915-8919.
41. Miller DG, Adam MA, Miller AD. Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection. Mol Cell Biol 1990; 10: 4239-4242.
42. Wang X-S, Yoder MC, Zhou SZ, Srivastava A. Parvovirus B19 promoter at map unit 6 confers autonomous replication-competence and erythroid-specificity to adeno-associated virus 2 in primary human hematopoietic progenitor cells. Proc Natl Acad Sci USA 1995; (in press).
43. Srivastava A. Replication of the adeno-associated virus DNA termini in vitro. Intervirology 1987; 27: 138-147.
44. Nahreini P, Srivastava A. Rescue and replication of the adeno-associated virus 2 genome in mortal and immortal human cells. Intervirology 1989; 30: 74-85.
45. Nahreini P, Srivastava A. Rescue of the adeno-associated virus 2 genome correlates with alterations in DNA-modifying enzymes in human cells. Intervirology 1992; 33: 109-115.
46. Samulski RJ, Chang L-S, Shenk TE. A recombinant plasmid from which an infectious adeno-associated virus genome can be excised in vitro and its use to study viral replication. J Virol 1987; 61: 3096-3101.
47. Samulski RJ, Chang L-S, Shenk TE. Helper-free stocks of recombinant adeno-associated viruses: normal integration does not require viral gene expression. J Virol 1989; 63: 3822-3828.
48. Cotmore SF, Tattersall P. Characterization and molecular cloning of a human parvovirus genome. Science 1984; 226: 1161-1165.
49. Nahreini P, Larsen SH, Srivastava A. Cloning and integration of DNA fragments in human cells via the inverted terminal repeats of the adeno-associated virus 2 genome. Gene 1992; 119: 265-272.
50. Nahreini P, Woody MJ, Zhou SZ, Srivastava A. Versatile adeno-associated virus 2-based vectors for constructing recombinant virions. Gene 1993; 124: 257-262.
51. Sambrook J, Fritsch EF, Maniatis T. Molecular Cloning. A Laboratory Manual. Cold Spring Harbor Press: Cold Spring Harbor, NY, 1989.
52. Zhou SZ et al. Adeno-associated virus 2-mediated gene transfer in murine hematopoietic progenitor cells. Exp Hematol 1993; 21: 928-933.
53. Ponnazhagan S, Nallar ML, Srivastava A. Suppression of human α-globin gene expression mediated by the recombinant adeno-associated virus 2-based antisense vectors. J Exp Med 1994; 179: 733-738.
54. Chomczynski P, Sacchi N. Single-step method of RNA isolation by acid guanidinium thiocyanate-phenol-chloroform extraction. Anal Biochem 1987; 162: 156-159.