A new aadnoviral helper-dependent vector results in long-term therapeutic levels of human coagulation factor IX at low doses in vivo

Blood

Volumn 99, Issue 11, 2002, Pages 3923-3930

  Ehrhardt, Anja ^a   Kay, Mark A ^a  

^a STANFORD UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; BLOOD CLOTTING FACTOR 9; INTERLEUKIN 6; TUMOR NECROSIS FACTOR ALPHA; RECOMBINANT PROTEIN;

ADENOVIRUS; ARTICLE; DNA SEQUENCE; DOSE RESPONSE; GENE EXPRESSION; GENE THERAPY; HELPER VIRUS; IMMUNE RESPONSE; PHENOTYPE; PRIORITY JOURNAL; TRANSGENE; ANIMAL; C57BL MOUSE; GENE VECTOR; GENETICS; HEMOPHILIA B; HUMAN; HUMAN ADENOVIRUS; INTRAVENOUS DRUG ADMINISTRATION; METHODOLOGY; MOUSE;

ADENOVIRUSES, HUMAN; ANIMALS; DOSE-RESPONSE RELATIONSHIP, DRUG; FACTOR IX; GENE THERAPY; GENETIC VECTORS; HELPER VIRUSES; HEMOPHILIA B; HUMANS; INFUSIONS, INTRAVENOUS; MICE; MICE, INBRED C57BL; RECOMBINANT PROTEINS;

EID: 0036592086     PISSN: 00064971     EISSN: None     Source Type: Journal    
DOI: 10.1182/blood.V99.11.3923     Document Type: Article

References (47)

1. Schiedner G, Morral N, Parks RJ, et al. Genomic DNA transfer with a high-capacity adenovirus vector results in improved in vivo gene expression and decreased toxicity. Nat Genet. 1998;18:180-183.
2. Balague C, Zhou J, Dai Y, et al. Sustained highlevel expression of full-length human factor VIII and restoration of clotting activity in hemophilic mice using a minimal adenovirus vector. Blood. 2000;95:820-828.
3. Morsy MA, Gu M, Motzel S, et al. An adenoviral vector deleted for all viral coding sequences results in enhanced safety and extended expression of a leptin transgene. Proc Natl Acad Sci U S A. 1998;95:7866-7871.
4. Wickham TJ, Roelvink PW, Brough DE, Kovesdi I. Adenovirus targeted to heparan-containing receptors increases its gene delivery efficiency to multiple cell types. Nat Biotechnol. 1996;14:1570-1573.
5. Kozarsky KF, Wilson JM. Gene therapy: Adenovirus vectors. Curr Opin Genet Dev. 1993;3:499-503.
6. Benihoud K, Yeh P, Perricaudet M. Adenovirus vectors for gene delivery. Curr Opin Biotechnol. 1999;10:440-447.
7. Stratford-Perricaudet LD, Levrero M, Chasse JF, Perricaudet M, Briand P. Evaluation of the transfer and expression in mice of an enzyme-encoding gene using a human adenovirus vector. Hum Gene Ther. 1990;1:241-256.
8. Parks RJ, Graham FL. A helper-dependent system for adenovirus vector production helps define a lower limit for efficient DNA packaging. J Virol. 1997;71:3293-3298.
9. Parks RJ, Chen L, Anton M, Sankar U, Rudnicki MA, Graham FL. A helper-dependent adenovirus vector system: Removal of helper virus by Cremediated excision of the viral packaging signal. Proc Natl Acad Sci U S A. 1996;93:13565-13570.
10. Parks RJ, Bramson JL, Wan Y, Addison CL, Graham FL. Effects of stuffer DNA on transgene expression from helper-dependent adenovirus vectors. J Virol. 1999;73:8027-8034.
11. Sandig V, Youil R, Bett AJ, et al. Optimization of the helper-dependent adenovirus system for production and potency in vivo. Proc Natl Acad Sci U S A. 2000;97:1002-1007.
12. Kalos M, Fournier RE. Position-independent transgene expression mediated by boundary elements from the apolipoprotein B chromatin domain. Mol Cell Biol. 1995;15:198-207.
13. Phi-Van L, von Kries JP, Ostertag W, Stratling WH. The chicken lysozyme 5′ matrix attachment region increases transcription from a heterologous promoter in heterologous cells and dampens position effects on the expression of transfected genes. Mol Cell Biol. 1990;10:2302-2307.
14. Krysan PJ, Calos MP. Epstein-Barrvirus-based vectors that replicate in rodent cells. Gene. 1993;136:137-143.
15. Krysan PJ, Smith JG, Calos MP. Autonomous replication in human cells of multimers of specific human and bacterial DNA sequences. Mol Cell Biol. 1993;13:2688-2696.
16. Kay MA, Meuse L, Gown AM, et al. Transient immunomodulation with anti-CD40 ligand antibody and CTLA4lg enhances persistence and secondary adenovirus-mediated gene transfer into mouse liver. Proc Natl Acad Sci U S A. 1997;94:4686-4691.
17. Kay MA, Landen CN, Rothenberg SR, et al. In vivo hepatic gene therapy: Complete albeit transient correction of factor IX deficiency in hemophilia B dogs. Proc Natl Acad Sci U S A. 1994;91:2353-2357.
18. Smith TA, Mehaffey MG, Kayda DB, et al. Adenovirus mediated expression of therapeutic plasma levels of human factor IX in mice. Nat Genet. 1993;5:397-402.
19. Miao CH, Ohashi K, Patijn GA, et al. Inclusion of the hepatic locus control region, an intron, and untranslated region increases and stabilizes hepatic factor IX gene expression in vivo but not in vitro. Mol Ther. 2000;1:522-532.
20. Hartigan-O'Connor D, Amalfitano A, Chamberlain JS. Improved production of gutted adenovirus in cells expressing adenovirus preterminal protein and DNA polymerase. J Virol. 1999;73:7835-7841.
21. Mizuguchi H, Kay MA. Efficient construction of a recombinant adenovirus vector by an improved in vitro ligation method. Hum Gene Ther. 1998;9:2577-2583.
22. Kay MA, Ponder KP, Woo SL. Human gene therapy: Present and future. Breast Cancer Res Treat. 1992;21:83-93.
23. Kay MA, Graham F, Leland F, Woo SL. Therapeutic serum concentrations of human alpha-1-antitrypsin after adenoviral-mediated gene transfer into mouse hepatocytes. Hepatology. 1995;21:815-819.
24. Lin HF, Maeda N, Smithies O, Straight DL, Stafford DW. A coagulation factor IX-deficient mouse model for human hemophilia B. Blood. 1997;90:3962-3966.
25. Peeters MJ, Patljn GA, Lieber A, Meuse L, Kay MA. Adenovirus-mediated hepatic gene transfer in mice: Comparison of Intravascular and biliary administration. Hum Gene Ther. 1996;7:1693-1699.
26. Lieber A, He CY, Meuse L, et al. The role of Kupffer cell activation and viral gene expression in early liver toxicity after infusion of recombinant adenovirus vectors. J Virol. 1997;71:8798-8807.
27. Miao CH, Thompson AR, Loeb K, Ye X. Longterm and therapeutic-level hepatic gene expression of human factor IX after naked plasmid transfer in vivo. Mol Ther. 2001;3:947-957.
28. Betz AG, Milstein C, Gonzalez-Fernandez A, Pannell R, Larson T, Neuberger MS. Elements regulating somatic hypermutation of an immunoglobulin kappa gene: Critical role for the intron enhancer/matrix attachment region. Cell. 1994;77:239-248.
29. Lozier JN, Metzger ME, Donahue RE, Morgan RA. Adenovirus-mediated expression of human coagulation factor IX in the rhesus macaque is associated with dose-limiting toxicity. Blood. 1999;94:3968-3975.
30. Morral N, O'Neal W, Rice K, et al. Administration of helper-dependent adenoviral vectors and sequential delivery of different vector serotype for long-term liver-directed gene transfer in baboons. Proc Natl Acad Sci U S A. 1999;96:12816-12821.
31. Bristol JA, Shirley P, Idamakanti N, Kaleko M, Connelly S. In vivo dose threshold effect of adenovirus-mediated factor VIII gene therapy in hemophiliac mice. Mol Ther. 2000;2:223-232.
32. Yang Y, Nunes FA, Berencsi K, Gonczol E, Engelhardt JF, Wilson JM. Inactivation of E2a in recombinant adenoviruses improves the prospect for gene therapy in cystic fibrosis. Nat Genet. 1994;7:362-369.
33. Yang Y, Nunes FA, Berencsi K, Furth EE, Gonczol E, Wilson JM. Cellular immunity to viral antigens limits E1-deleted adenoviruses for gene therapy. Proc Natl Acad Sci U S A. 1994;91:4407-4411.
34. Dong JY, Wang D, Van Ginkel FW, Pascual DW, Frizzell RA. Systematic analysis of repeated gene delivery into animal lungs with a recombinant adenovirus vector. Hum Gene Ther. 1996;7:319-331.
35. Engelhardt JF, Simon RH, Yang Y, et al. Adenovirus-mediated transfer of the CFTR gene to lung of nonhuman primates: Biological efficacy study. Hum Gene Ther. 1993;4:759-769.
36. Wolff G, Worgall S, van Rooijen N, Song WR, Harvey BG, Crystal RG. Enhancement of in vivo adenovirus-mediated gene transfer and expression by prior depletion of tissue macrophages in the target organ. J Virol. 1997;71:624-629.
37. Schnell MA, Zhang Y, Tazelaar J, et al. Activation of innate immunity in nonhuman primates following intraportal administration of adenoviral vectors. Mol Ther. 2001;3:708-722.
38. Tao N, Gao GP, Parr M, et al. Sequestration of adenoviral vector by Kupffer cells leads to a nonlinear dose response of transduction in liver. Mol Ther. 2001;3:28-35.
39. Gooding LR. Regulation of TNF-mediated cell death and inflammation by human adenoviruses. Infect Agents Dis. 1994;3:106-115.
40. Noah TL, Wortman IA, Hu PC, Leigh MW, Boucher RC. Cytokine production by cultured human bronchial epithelial cells infected with a replication-deficient adenoviral gene transfer vector or wild-type adenovirus type 5. Am J Respir Cell Mol Biol. 1996;14:417-424.
41. Ginsberg HS, Moldawer LL, Sehgal PB, et al. A mouse model for investigating the molecular pathogenesis of adenovirus pneumonia. Proc Natl Acad Sci U S A. 1991;88:1651-1655.
42. Fields PA, Arruda VR, Armstrong E, et al. Risk and prevention of anti-factor IX formation in AAV-mediated gene transfer in the context of a large deletion of F9. Mol Ther. 2001;4:201-210.
43. Ge Y, Powell S, Van Roey M, McArthur JG. Factors influencing the development of an anti-factor IX (FIX) immune response following administration of adeno-associated virus-FIX. Blood. 2001;97:3733-3737.
44. Lillicrap D. Hemophilia treatment. Gene therapy, factor VIII antibodies and immune tolerance: hopes and concerns [discussion appears in Haematologica. 2000;85:111-102]. Haematologica. 2000;85:108-111.
45. Pastore L, Morral N, Zhou H, et al. Use of a liverspecific promoter reduces immune response to the transgene in adenoviral vectors. Hum Gene Ther. 1999;10:1773-1781.
46. Barr D, Tubb J, Ferguson D, et al. Strain related variations in adenovirally mediated transgene expression from mouse hepatocytes in vivo: Comparisons between immunocompetent and immunodeficient inbred strains. Gene Ther. 1995;2:151-155.
47. Schowalter DB, Himeda CL, Winther BL, Wilson CB, Kay MA. Implication of interfering antibody formation and apoptosis as two different mechanisms leading to variable duration of adenovirus-mediated transgene expression in immune-competent mice. J Virol. 1999;73:4755-4766.