Transduction of primitive human marrow and cord blood-derived hematopoietic progenitor cells with adeno-associated virus vectors

Blood

Volumn 93, Issue 6, 1999, Pages 1882-1894

  Chatterjee, Saswati ^a,b   Li, Wei ^b   Wong, Christie Ann ^b   Fisher Adams, Grace ^b   Lu, Di ^b   Guha, Mausumee ^b   Macer, James A ^b   Forman, Stephen J ^b   Wong Jr , K K ^b  

^a CITY OF HOPE NATIONAL MEDICAL CENTER   (United States)

^b NONE

Author keywords

[No Author keywords available]

Indexed keywords

ALKALINE PHOSPHATASE; CD34 ANTIGEN; VIRUS VECTOR;

ARTICLE; BONE MARROW CELL; FLUORESCENCE IN SITU HYBRIDIZATION; GENE THERAPY; GENE TRANSFER; GENETIC TRANSCRIPTION; GENETIC TRANSDUCTION; HUMAN; HUMAN TISSUE; INTERPHASE; METAPHASE; PRIORITY JOURNAL; PROTEIN EXPRESSION; STEM CELL; UMBILICAL CORD BLOOD;

EID: 0033559749     PISSN: 00064971     EISSN: None     Source Type: Journal    
DOI: 10.1182/blood.v93.6.1882.406k03_1882_1894     Document Type: Article

References (55)

1. Blaese RM, Culver KW, Miller AD, Carter CS, Fleisher T, Clerici M, Shearer G, Chang L, Chiang, Tolstoshev P, Greenblatt J, Rosenberg S, Klein H, Berger M, Mullen C, Ramsey W, Muul L, Morgan R, Anderson WF: T lymphocyte-directed gene therapy for ADA-SCID: Initial trial results after 4 years. Science 270:475, 1995
2. Kohn DB, Weinberg K, Nolta J, Heiss L, Lenarsky C, Crooks G, Hanley M, Annett G, Brooks J, El-Khoureiy A, Lawrence K, Wells S, Moen R, Bastian J, Williams-Herman D, Elder M, Wara D, Bowen T, Hershfiald M, Mullen C, Blaese M, Parkman R: Engraftment of gene-modified umbilical cord blood cells in neonates with adenosine deaminase deficiency. Nat Med 1:1017, 1995
3. Helene C: Control of oncogene expression by antisense nucleic acids. Eur J Cancer 30A:1721, 1994
4. Shaughnessy E, Lu D, Chatterjee S, Wong KK: Parvoviral vectors for the gene therapy of cancer. Semin Oncol 23:159, 1995
5. Chatterjee S, Johnson PR, Wong KK: Dual-target inhibition of HIV-1 in vitro by means of an adeno-associated virus antisense vector. Science 258:1485, 1992
6. Brenner MK, Rill DR, Moen RC, Krance, RA, Mirro J, Anderson WF, Ihle JN: Gene-marking to trace origin of relapse after autologous bone-marrow transplantation. Lancet 341:85, 1993
7. + cells present in autologous transplants of chronic myelogenous leukemia (CML) contribute to relapse after autologous bone marrow in CML. Blood 10:3068, 1994
8. Dunbar CE, Cottler-Fox M, O'Shaughnessy J, Doren S, Carter C, Berenson R, Brown S, Moen R, Greenblatt J, Stewart M, Leitman S, Wilson W, Cowan K, Young N, Nienhuis A: Retrovirally marked CD34-enriched peripheral blood and bone marrow cells contribute to long term engraftment after autologous transplantation. Blood 85:3048, 1995
9. Larochelle A, Vormoor J, Hanenberg H, Wang JC, Bhatia M, Lapidot T, Moritz T, Murdoch B, Xiao XL, Kato I, Williams DA, Dick JE: Identification of primitive human hematopoietic cells capable of repopulating NOD/SCID mouse bone marrow: Implications for gene therapy. Nat Med 2:1329, 1996
10. Huang MM, Wong A, Yu X, Kakkis E, Kohn DB: Retrovirus mediated transfer of the human alpha-L-iduronidase cDNA into human hematopoietic progenitor cells leads to the correction in trans of Hurler fibroblasts. Gene Ther 4:1150, 1997
11. 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 10:4239, 1990
12. Roe TY, Reynolds TC, Yu G, Brown PO: Integration of murine leukemia virus DNA depends on mitosis. EMBO J 12:2099, 1993
13. Spangrude GJ, Johnson GR: Resting and activated subsets of mouse multipotent hematopoietic stem cells. Proc Natl Acad Sci USA 87:7433, 1990
14. Muzyczka N: Use of AAV as a general transduction vector for mammalian cells. Curr Top Microbiol Immunol 158:97, 1992
15. Berns KI, Giraud C: Biology of adeno-associated virus. Curr Top Microbiol Immunol 218:1, 1996
16. Janik JE, Huston MM, Rose JA: Locations of adenovirus genes required for the replication of adeno-associated virus. Proc Natl Acad Sci USA 78:1925, 1981
17. Buller RML, Janik J, Sebring ED, Rose JA: Herpes simplex virus types 1 and 2 completely help adenovirus-associated virus replication. J Virol 40:241, 1981
18. Kotin RM, Siniscalco M, Samulski RJ, Zhu XD, Hunter L, Laughlin CA, McLaughlin S, Muzyczka N, Rocchi M, Berns KI: Site-specific integration by adeno-associated virus. Proc Natl Acad Sci USA 87:2211, 1990
19. Samulski RJ, Zhu X, Xiao X, Brook JD, Housman DE, Epstein N, Hunter LA: Targeted integration of adeno-associated virus (AAV) into human chromosome 19. EMBO J 10:3941, 1991
20. Hermonat PL, Muzyczka N: Use of adeno-associated virus as a mammalian DNA cloning vector: Transduction of neomycin resistance into mammalian tissue culture cells. Proc Natl Acad Sci USA 81:6466, 1984
21. Bertrand E, Castanotto D, Zhou C, Carbonelle C, Lee NS, Good P, Chatterjee S, Grange T, Pictet R, Kohn D, Engelke D, Rossi J: The expression cassette determines the functional activity of ribozymes in mammalian cells by controlling their intracellular localization. RNA 3:75, 1997
22. Podsakoff G, Wong KK Jr, Chatterjee S: Stable and efficient gene transfer into non-dividing cells by adeno-associated virus (AAV)-based vectors. J Virol 68:5656, 1994
23. Kaplitt MG, Leone P, Samulski RJ, Xiao X, Pfaff DW, O'Malley KL, During MJ: Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain. Nat Genet 8:148, 1994
24. Du B, Wu P, Boldt-Houle DM, Terwilliger EF: Efficient transduction of human neurons with an adeno-associated virus vector. Gene Ther 3:254, 1996
25. Kaplitt MG, Xiao X, Samulski RJ, Li J, Ojamaa K, Klein IL, Makimura H, Kaplitt MJ, Strumpf RK, Diethrich EB: Long-term gene transfer in porcine myocardium after coronary infusion of an adeno-associated virus vector. Ann Thorac Surg 62:1669, 1996
26. Xiao X, Li J, Samulski RJ: Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector. J Virol 70:8098, 1996
27. Kessler PD, Podsakoff GM, Chen X, McQuiston SA, Colosi PC, Matelis LA, Kurtzman GJ, Byrne BJ: Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein. Proc Natl Acad Sci USA 93:14082, 1996
28. Fisher-Adams G, Wong KK, Podsakoff GM, Forman S, Chatterjee S: Integration of adeno-associated virus vector genomes in human CD34 cells following transduction. Blood 88:492, 1996
29. Zhou SZ, Cooper S, Kang LY, Ruggieri L, Heimfeld S, Srivastava A, Broxmeyer HE: 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 179:1867, 1994
30. Miller JL, Donahue RE, Sellers SE, Samulski RJ, Young NS, Nienhuis AW: Recombinant adeno-associated virus (rAAV)-mediated expression of a human gamma-globin gene in human progenitor-derived erythroid cells. Proc Natl Acad Sci USA 91:10183, 1994
31. Walsh CE, Nienhuis AW, Samulski RJ, Brown MG, Miller JL, Young NS, Liu JM: Phenotypic correction of Fanconi anemia in human hematopoietic cells with a recombinant adeno-associated virus vector. J Clin Invest 94:1440, 1994
32. Luhovy M, McCune S, Dong JY, Prchal JF, Townes TM, Prchal JT: Stable transduction of recombinant adeno-associated virus into hematopoietic stem cells from normal and sickle cell patients. Biol Blood Marrow Transplant 2:24, 1996
33. Ponnazhagan S, Srivastava A, Wadsworth S, Kaplan Zhou SZ, Wang XS, Yoder MC, Mukherjee P: Adeno-associated virus 2-mediated gene transfer in vivo: organ-tropism and expression of transduced sequences in mice. Gene 190:203, 1997
34. Ponnazhagan S, Srivastava A, Yoder MC: Adeno-associated virus type 2-mediated transduction of murine hematopoietic cells with long-term repopulating ability and sustained expression of a human globin gene in vivo. J Virol 71:3098, 1997
35. Ezra E, Blacher R, Udenfiend S: Purification and partial sequencing of human placental alkaline phosphatase. Biochem Biophys Res Commun 116:1076, 1983
36. Fields-Berry SC, Halliday AL, Cepko CL: A recombinant retrovirus encoding alkaline phosphatase confirms clonal boundary assignment in lineage analysis of murine retina. Proc Natl Acad Sci USA 89:693, 1992
37. Chatterjee S, Wong KK: Adeno-associated viral vectors for the delivery of antisense RNA. Methods: A Companion to Methods in Enzymology 5:51, 1993
38. Ausubel FM, Brent R, Kingston RE, Moore D, Seidman JG, Smith JA, Struhl K: Short Protocols in Molecular Biology. New York, NY, Wiley, 1992
39. Dexter TM, Allen TD, Lajtha LG: Conditions controlling the proliferation of haemopoietic stem cells in vitro. J Cell Physiol 91:335, 1977
40. Sutherland HJ, Eaves CJ, Eaves AC, Dragowska W, Lansdorp PM: Characterization and partial purification of human marrow cells capable of initiating long-term hematopoiesis in vitro. Blood 74:1563, 1989
41. Detection and Counterstaining Procedure, in Oncor: Oncor Chromosome In Situ Hybridization System Handbook. 1994, p 20
42. Wonderling RS, Owens RAJ: Binding sites for adeno-associated virus Rep proteins within the human genome. J Virol 71:2528, 1997
43. Linden RM, Berns KI, Winocour E: The recombination signals for adeno-associated virus site-specific integration. Proc Natl Acad Sci USA 93:7966, 1996
44. Ryan JH, Muzyczka N, Zolotukhin S: Sequence requirements for binding of Rep68 to the adeno-associated virus terminal repeats. J Virol 70:1542, 1996
45. Im DS, Muzyczka N: The AAV origin binding protein Rep68 is an ATP-dependent site-specific endonucleadse with DNA helicase activity. Cell 61:447, 1990
46. Yang CC, Xiao X, Zhu X, Ansardi DC, Epstein ND, Frey MR, Matera AG, Samulski RJ: Cellular recombination pathways and viral terminal repeat hairpin structures are sufficient for adeno-associated virus integration in vivo and in vitro. J Virol 71:9231, 1997
47. Ponnazhagan S, Srivastava A, Wang XS, Nahreini P, Zhou SZ, Kearns WG, Erikson D: Lack of site-specific integration of the recombinant adeno-associated virus 2 genomes in human cells. Hum Gene Ther 8:275, 1997
48. Duan D, Engelhardt JF, Burda JF, Fisher KJ: Structural and functional heterogeneity of integrated recombinant AAV genomes. Virus Res 48:41, 1997
49. Ponnazhagan S, Srivastava A, Srour EF, Yoder MC, Kurpad C, Mah C, Kube DM, Qing K, Wang XS, Mukherjee P: Adeno-associated virus type 2-mediated transduction in primary human bone marrow-derived CD34+ hematopoietic progenitor cells: Donor variation and correlation of transgene expression with cellular differentiation. J Virol 71:8262, 1997
50. Gardner JP, Scadden DT, Kurtzman GJ, Colosi PC, Zhu H: Robust, but transient expression of adeno-associated virus-transduced genes during human T lymphopoiesis. Blood 90:4854, 1997
51. Mizukami H, Young NS, Brown KE: Adeno-associated virus type 2 binds to a 150 kilodalton cell membrane glycoprotein. Virology 217:124, 1996
52. Summerford C, Samulski RJ: Membrane associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions. J Virol 72:1438, 1998
53. Qing K, Srivastava A, Bajpai A, Ponnazhagan S, Kube DM, Wang XS: Role of tyrosine phosphorylation of a cellular protein in adeno-associated virus 2-mediated transgene expression. Proc Natl Acad Sci USA 94:10879, 1997
54. McCown TJ, Xiao X, Li J, Breese GR, Samulski RJ: Differential and persistent expression patterns of CNS gene transfer by an adeno-associated virus vector. Brain Res 713:99, 1996
55. Flotte T: Presented at the Cystic Fibrosis Foundation Williamsburg Gene Therapy Conference, June 1997