Adenoviral vectors for transient gene expression in human primitive hematopoietic cells: Applications and prospects

Experimental Hematology

Volumn 35, Issue 3, 2007, Pages 343-349

  Järås, Marcus ^a   Brun, Ann C M ^a   Karlsson, Stefan ^a   Fan, Xiaolong ^a,b  

^a Lund Strategic Research Center for Stem Cell Biology and Cell Therapy   (Sweden)

^b LUND UNIVERSITY   (Sweden)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR;

BONE MARROW CELL; CD34 SELECTION; CELL DIFFERENTIATION; CELL DIVISION; CELL PROLIFERATION; GENE EXPRESSION; GENE TRANSFER; HEMATOPOIETIC CELL; HUMAN; LEUKEMIA CELL; MUTAGENESIS; NONHUMAN; PRIORITY JOURNAL; REVIEW; SIGNAL TRANSDUCTION;

ADENOVIRIDAE; ANIMALS; GENE EXPRESSION; GENE TRANSFER TECHNIQUES; GENES, REPORTER; GENETIC VECTORS; HEMATOPOIETIC STEM CELLS; HUMANS;

EID: 33847037957     PISSN: 0301472X     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.exphem.2006.11.004     Document Type: Review

References (63)

1. Hacein-Bey-Abina S., Von Kalle C., Schmidt M., et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 302 (2003) 415-419
2. Kustikova O., Fehse B., Modlich U., et al. Clonal dominance of hematopoietic stem cells triggered by retroviral gene marking. Science 308 (2005) 1171-1174
3. Li Z., Dullmann J., Schiedlmeier B., et al. Murine leukemia induced by retroviral gene marking. Science 296 (2002) 497
4. Shayakhmetov D.M., Papayannopoulou T., Stamatoyannopoulos G., and Lieber A. Efficient gene transfer into human CD34(+) cells by a retargeted adenovirus vector. J Virol 74 (2000) 2567-2583
5. Yotnda P., Onishi H., Heslop H.E., et al. Efficient infection of primitive hematopoietic stem cells by modified adenovirus. Gene Ther 8 (2001) 930-937
6. Knaan-Shanzer S., Van Der Velde I., Havenga M.J., Lemckert A.A., De Vries A.A., and Valerio D. Highly efficient targeted transduction of undifferentiated human hematopoietic cells by adenoviral vectors displaying fiber knobs of subgroup B. Hum Gene Ther 12 (2001) 1989-2005
7. Nilsson M., Karlsson S., and Fan X. Functionally distinct subpopulations of cord blood CD34+ cells are transduced by adenoviral vectors with serotype 5 or 35 tropism. Mol Ther 9 (2004) 377-388
8. Jaras M., Edqvist A., Rebetz J., Salford L.G., Widegren B., and Fan X. Human short-term repopulating cells have enhanced telomerase reverse transcriptase expression. Blood 108 (2006) 1084-1091
9. Wattel E., Vanrumbeke M., Abina M.A., et al. Differential efficacy of adenoviral mediated gene transfer into cells from hematological cell lines and fresh hematological malignancies. Leukemia 10 (1996) 171-174
10. Rebel V.I., Hartnett S., Denham J., Chan M., Finberg R., and Sieff C.A. Maturation and lineage-specific expression of the coxsackie and adenovirus receptor in hematopoietic cells. Stem Cells 18 (2000) 176-182
11. Neering S.J., Hardy S.F., Minamoto D., Spratt S.K., and Jordan C.T. Transduction of primitive human hematopoietic cells with recombinant adenovirus vectors. Blood 88 (1996) 1147-1155
12. MacKenzie K.L., Hackett N.R., Crystal R.G., and Moore M.A. Adenoviral vector-mediated gene transfer to primitive human hematopoietic progenitor cells: assessment of transduction and toxicity in long-term culture. Blood 96 (2000) 100-108
13. Takahashi T., Yamada K., Tanaka T., et al. A potential molecular approach to ex vivo hematopoietic expansion with recombinant epidermal growth factor receptor-expressing adenovirus vector. Blood 91 (1998) 4509-4515
14. Douglas J.T., Rogers B.E., Rosenfeld M.E., Michael S.I., Feng M., and Curiel D.T. Targeted gene delivery by tropism-modified adenoviral vectors. Nat Biotechnol 14 (1996) 1574-1578
15. Miller C.R., Buchsbaum D.J., Reynolds P.N., et al. Differential susceptibility of primary and established human glioma cells to adenovirus infection: targeting via the epidermal growth factor receptor achieves fiber receptor-independent gene transfer. Cancer Res 58 (1998) 5738-5748
16. Hong S.S., Galaup A., Peytavi R., Chazal N., and Boulanger P. Enhancement of adenovirus-mediated gene delivery by use of an oligopeptide with dual binding specificity. Hum Gene Ther 10 (1999) 2577-2586
17. Marini III F.C., Yu Q., Wickham T., Kovesdi I., and Andreeff M. Adenovirus as a gene therapy vector for hematopoietic cells. Cancer Gene Ther 7 (2000) 816-825
18. Wickham T.J., Tzeng E., Shears II L.L., et al. Increased in vitro and in vivo gene transfer by adenovirus vectors containing chimeric fiber proteins. J Virol 71 (1997) 8221-8229
19. Dmitriev I., Krasnykh V., Miller C.R., et al. An adenovirus vector with genetically modified fibers demonstrates expanded tropism via utilization of a coxsackievirus and adenovirus receptor-independent cell entry mechanism. J Virol 72 (1998) 9706-9713
20. Wu H., Seki T., Dmitriev I., Uil T., Kashentseva E., Han T., and Curiel D.T. Double modification of adenovirus fiber with RGD and polylysine motifs improves coxsackievirus-adenovirus receptor-independent gene transfer efficiency. Hum Gene Ther 13 (2002) 1647-1653
21. Belousova N., Korokhov N., Krendelshchikova V., et al. Genetically targeted adenovirus vector directed to CD40-expressing cells. J Virol 77 (2003) 11367-11377
22. Stevenson S.C., Rollence M., Marshall-Neff J., and McClelland A. Selective targeting of human cells by a chimeric adenovirus vector containing a modified fiber protein. J Virol 71 (1997) 4782-4790
23. Nilsson M., Ljungberg J., Richter J., et al. Development of an adenoviral vector system with adenovirus serotype 35 tropism; efficient transient gene transfer into primary malignant hematopoietic cells. J Gene Med 6 (2004) 631-641
24. Fan X., Brun A., Segren S., Jacobsen S.E., and Karlsson S. Efficient adenoviral vector transduction of human hematopoietic SCID-repopulating and long-term culture-initiating cells. Hum Gene Ther 11 (2000) 1313-1327
25. Byk T., Haddada H., Vainchenker W., and Louache F. Lipofectamine and related cationic lipids strongly improve adenoviral infection efficiency of primitive human hematopoietic cells. Hum Gene Ther 9 (1998) 2493-2502
26. Howard D.S., Rizzierri D.A., Grimes B., et al. Genetic manipulation of primitive leukemic and normal hematopoietic cells using a novel method of adenovirus-mediated gene transfer. Leukemia 13 (1999) 1608-1616
27. Kitazono M., Rao V.K., Robey R., et al. Histone deacetylase inhibitor FR901228 enhances adenovirus infection of hematopoietic cells. Blood 99 (2002) 2248-2251
28. Gaggar A., Shayakhmetov D.M., and Lieber A. CD46 is a cellular receptor for group B adenoviruses. Nat Med 9 (2003) 1408-1412
29. Segerman A., Atkinson J.P., Marttila M., Dennerquist V., Wadell G., and Arnberg N. Adenovirus type 11 uses CD46 as a cellular receptor. J Virol 77 (2003) 9183-9191
30. Sakurai F., Mizuguchi H., and Hayakawa T. Efficient gene transfer into human CD34+ cells by an adenovirus type 35 vector. Gene Ther 10 (2003) 1041-1048
31. Vogels R., Zuijdgeest D., van Rijnsoever R., et al. Replication-deficient human adenovirus type 35 vectors for gene transfer and vaccination: efficient human cell infection and bypass of preexisting adenovirus immunity. J Virol 77 (2003) 8263-8271
32. Dipaolo N., Ni S., Gaggar A., et al. Evaluation of adenovirus vectors containing serotype 35 fibers for vaccination. Mol Ther 13 (2006) 756-765
33. Ni S., Bernt K., Gaggar A., Li Z.Y., Kiem H.P., and Lieber A. Evaluation of biodistribution and safety of adenovirus vectors containing group B fibers after intravenous injection into baboons. Hum Gene Ther 16 (2005) 664-677
34. He T.C., Zhou S., da Costa L.T., Yu J., Kinzler K.W., and Vogelstein B. A simplified system for generating recombinant adenoviruses. Proc Natl Acad Sci U S A 95 (1998) 2509-2514
35. Wang H., Shayakhmetov D.M., Leege T., et al. A capsid-modified helper-dependent adenovirus vector containing the beta-globin locus control region displays a nonrandom integration pattern and allows stable, erythroid-specific gene expression. J Virol 79 (2005) 10999-11013
36. Fan X., Brun A., and Karlsson S. Adenoviral vector design for high-level transgene expression in primitive human hematopoietic progenitors. Gene Ther 7 (2000) 2132-2138
37. Sakurai F., Kawabata K., Yamaguchi T., Hayakawa T., and Mizuguchi H. Optimization of adenovirus serotype 35 vectors for efficient transduction in human hematopoietic progenitors: comparison of promoter activities. Gene Ther 12 (2005) 1424-1433
38. Fan X., Valdimarsdottir G., Larsson J., et al. Transient disruption of autocrine TGF-beta signaling leads to enhanced survival and proliferation potential in single primitive human hemopoietic progenitor cells. J Immunol 168 (2002) 755-762
39. Brun A.C., Fan X., Bjornsson J.M., Humphries R.K., and Karlsson S. Enforced adenoviral vector-mediated expression of HOXB4 in human umbilical cord blood CD34+ cells promotes myeloid differentiation but not proliferation. Mol Ther 8 (2003) 618-628
40. Frey B.M., Hackett N.R., Bergelson J.M., et al. High-efficiency gene transfer into ex vivo expanded human hematopoietic progenitors and precursor cells by adenovirus vectors. Blood 91 (1998) 2781-2792
41. Borge O.J., Ramsfjell V., Cui L., and Jacobsen S.E. Ability of early acting cytokines to directly promote survival and suppress apoptosis of human primitive CD34+CD38- bone marrow cells with multilineage potential at the single-cell level: key role of thrombopoietin. Blood 90 (1997) 2282-2292
42. Ramsfjell V., Borge O.J., Cui L., and Jacobsen S.E. Thrombopoietin directly and potently stimulates multilineage growth and progenitor cell expansion from primitive (CD34+ CD38-) human bone marrow progenitor cells: distinct and key interactions with the ligands for c-kit and flt3, and inhibitory effects of TGF-beta and TNF-alpha. J Immunol 158 (1997) 5169-5177
43. Buske C., Feuring-Buske M., Abramovich C., et al. Deregulated expression of HOXB4 enhances the primitive growth activity of human hematopoietic cells. Blood 100 (2002) 862-868
44. Weissman IL. Stem cells: units of development, units of regeneration, and units in evolution. Cell. 2000;100:157-168.
45. Ko D., Hawkins L., and Yu D.C. Development of transcriptionally regulated oncolytic adenoviruses. Oncogene 24 (2005) 7763-7774
46. Li Y., Yu D.C., Chen Y., et al. A hepatocellular carcinoma-specific adenovirus variant, CV890, eliminates distant human liver tumors in combination with doxorubicin. Cancer Res 61 (2001) 6428-6436
47. Li X., Zhao X., Fang Y., et al. Generation of destabilized green fluorescent protein as a transcription reporter. J Biol Chem 273 (1998) 34970-34975
48. Edqvist A., Rebetz J., Jaras M., et al. Detection of cell cycle- and differentiation stage-dependent human telomerase reverse transcriptase expression in single living cancer cells. Mol Ther 14 (2006) 139-148
49. Engelhardt M., Kumar R., Albanell J., Pettengell R., Han W., and Moore M.A. Telomerase regulation, cell cycle, and telomere stability in primitive hematopoietic cells. Blood 90 (1997) 182-193
50. Yui J., Chiu C.P., and Lansdorp P.M. Telomerase activity in candidate stem cells from fetal liver and adult bone marrow. Blood 91 (1998) 3255-3262
51. Marrone A., Stevens D., Vulliamy T., Dokal I., and Mason P.J. Heterozygous telomerase RNA mutations found in dyskeratosis congenita and aplastic anemia reduce telomerase activity via haploinsufficiency. Blood 104 (2004) 3936-3942
52. Vulliamy T., Marrone A., Szydlo R., Walne A., Mason P.J., and Dokal I. Disease anticipation is associated with progressive telomere shortening in families with dyskeratosis congenita due to mutations in TERC. Nat Genet 36 (2004) 447-449
53. Vulliamy T., Marrone A., Dokal I., and Mason P.J. Association between aplastic anaemia and mutations in telomerase RNA. Lancet 359 (2002) 2168-2170
54. Vulliamy T., Marrone A., Goldman F., et al. The RNA component of telomerase is mutated in autosomal dominant dyskeratosis congenita. Nature 413 (2001) 432-435
55. Fogarty P.F., Yamaguchi H., Wiestner A., et al. Late presentation of dyskeratosis congenita as apparently acquired aplastic anaemia due to mutations in telomerase RNA. Lancet 362 (2003) 1628-1630
56. Yamaguchi H., Baerlocher G.M., Lansdorp P.M., et al. Mutations of the human telomerase RNA gene (TERC) in aplastic anemia and myelodysplastic syndrome. Blood 102 (2003) 916-918
57. Allsopp R.C., Morin G.B., DePinho R., Harley C.B., and Weissman I.L. Telomerase is required to slow telomere shortening and extend replicative lifespan of HSCs during serial transplantation. Blood 102 (2003) 517-520
58. Allsopp R.C., Morin G.B., Horner J.W., DePinho R., Harley C.B., and Weissman I.L. Effect of TERT over-expression on the long-term transplantation capacity of hematopoietic stem cells. Nat Med 9 (2003) 369-371
59. Chung J.H., Bell A.C., and Felsenfeld G. Characterization of the chicken beta-globin insulator. Proc Natl Acad Sci U S A 94 (1997) 575-580
60. Steinwaerder D.S., and Lieber A. Insulation from viral transcriptional regulatory elements improves inducible transgene expression from adenovirus vectors in vitro and in vivo. Gene Ther 7 (2000) 556-567
61. Hock H., Hamblen M.J., Rooke H.M., et al. Gfi-1 restricts proliferation and preserves functional integrity of haematopoietic stem cells. Nature 431 (2004) 1002-1007
62. Park I.K., Qian D., Kiel M., et al. Bmi-1 is required for maintenance of adult self-renewing haematopoietic stem cells. Nature 423 (2003) 302-305
63. Zeng H., Yucel R., Kosan C., Klein-Hitpass L., and Moroy T. Transcription factor Gfi1 regulates self-renewal and engraftment of hematopoietic stem cells. EMBO J 23 (2004) 4116-4125