Use of multiply deleted adenovirus vectors to probe adenovirus vector performance and toxicities

Current Opinion in Molecular Therapeutics

Volumn 5, Issue 4, 2003, Pages 362-366

  Amalfitano, Andrea ^a  

^a DUKE UNIVERSITY MEDICAL CENTER   (United States)

Author keywords

Adenovirus vector; Gene therapy; Multiply deleted adenovirus; Toxicity

Indexed keywords

ADENOVIRUS VECTOR; ALPHA 1 ANTITRYPSIN; BETA GALACTOSIDASE; CAPSID PROTEIN; DNA POLYMERASE; SINGLE STRANDED DNA BINDING PROTEIN;

CYTOTOXICITY; GENE DELETION; GENE EXPRESSION; GENE FUNCTION; GENETIC TRANSDUCTION; HUMAN; IMMUNE RESPONSE; IMMUNOGENICITY; LIVER TOXICITY; MEDICAL RESEARCH; NONHUMAN; OTOTOXICITY; REVIEW; VIRAL GENE DELIVERY SYSTEM; VIRUS ATTENUATION; VIRUS GENOME; VIRUS REPLICATION;

ADENOVIRIDAE; ANIMALS; GENE THERAPY; GENETIC VECTORS; HUMANS;

ADENOVIRIDAE; ANIMALIA; EQUUS CABALLUS;

EID: 0042326379     PISSN: 14648431     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Review

References (49)

1. Thomas GP, Mathews MP: DNA replication and the early to late transition in adenovirus infection. Cell (1980) 22(2 Pt 2):523-533.
2. Imperiale MJ: Molecular biology of adenovirus gene therapy vectors. In: Viral Vectors: Basic Science and Gene Therapy. Cid-Arregui A, García Carrancá A (Eds), BioTechniques Press, Westborough, MA, USA (2001):119-128.
3. Graham FL, Smiley J, Russell WC, Nairn R: Characteristics of a human cell line transformed by DNA from human adenovirus type 5. J Gen Virol (1977) 36(1):59-74.
4. Barr D, Tubb J, Ferguson D, Scaria A, Lieber A, Wilson C, Perkins J, Kay MA: Strain related variations in adenovirally mediated transgene expression from mouse hepatocytes in vivo: Comparisons between immunocompetent and immunodeficient inbred strains. Gene Ther (1995) 2(2):151-155.
5. De Geest B, Van Linthout S, Lox M, Collen D, Holvoet P: Sustained expression of human apolipoprotein A-I after adenoviral gene transfer in C57BL/6 mice: Role of apolipoprotein A-I promoter, apolipoprotein A-I introns, and human apolipoprotein E enhancer. Hum Gene Ther (2000) 11(1):101-112.
6. Gao GP, Yang Y, Wilson JM: Biology of adenovirus vectors with E1 and E4 deletions for liver-directed gene therapy. J Virol (1996) 70(12):8934-8943.
7. Guo ZS, Wang LH, Eisensmith RC, Woo SL: Evaluation of promoter strength for hepatic gene expression in vivo following adenovirus-mediated gene transfer. Gene Ther (1996) 3(9):802-810.
8. Holt KH, Lim LE, Straub V, Venzke DP, Duclos F, Anderson RD, Davidson BL, Campbell KP: Functional rescue of the sarcoglycan complex in the BIO 14.6 hamster using δ-sarcoglycan gene transfer. Mol Cell (1998) 1(6):841-848.
9. Kozarsky KF, Jooss K, Donahee M, Strauss JF, Wilson JM: Effective treatment of familial hypercholesterolaemia in the mouse model using adenovirus-mediated transfer of the VLDL receptor gene. Nat Genet (1996) 13(1):54-62.
10. Michou AI, Santoro L, Christ M, Julliard V, Pavirani A, Mehtali M: Adenovirus-mediated gene transfer: Influence of transgene, mouse strain and type of immune response on persistence of transgene expression. Gene Ther (1997) 4(5):473-482.
11. Scaria A, St George JA, Jiang C, Kaplan JM, Wadsworth SC, Gregory RJ: Adenovirus-mediated persistent cystic fibrosis transmembrane conductance regulator expression in mouse airway epithelium. J Virol (1998) 72(9):7302-7309.
12. Svensson EC, Black HB, Dugger DL, Tripathy SK, Goldwasser E, Hao Z, Chu L, Leiden JM: Long-term erythropoletin expression in rodents and non-human primates following intramuscular injection of a replication-defective adenoviral vector. Hum Gene Ther (1997) 8(15):1797-1806.
13. Tripathy SK, Svensson EC, Black HB, Goldwasser E, Margalith M, Hobart PM, Leiden JM: Long-term expression of erythropoietin in the systemic circulation of mice after intramuscular injection of a plasmid DNA vector. Proc Natl Acad Sci USA (1996) 93(20):10876-10880.
14. Ziegler RJ, Yew NS, Li C, Cherry M, Berthelette P, Romanczuk H, Ioannou YA, Zeidner KM, Desnick RJ, Cheng SH: Correction of enzymatic and lysosomal storage defects in Fabry mice by adenovirus-mediated gene transfer. Hum Gene Ther(1999) 10(10):1667-1682.
15. Zoltick PW, Chirmule N, Schnell MA, Gao GP, Hughes JV, Wilson JM: Biology of E1-deleted adenovirus vectors in nonhuman primate muscle. J Virol (2001) 75(11):5222-5229.
16. Kass-Eisler A, Leinwand L, Gall J, Bloom B, Falck-Pedersen E: Circumventing the immune response to adenovirus-mediated gene therapy. Gene Ther (1996) 3(2):154-162.
17. Mack CA, Song WR, Carpenter H, Wickham TJ, Kovesdi I, Harvey BG, Magovern CJ, Isom OW, Rosengart T, Falck-Pedersen E, Hackett NR et al: Circumvention of anti-adenovirus neutralizing immunity by administration of an adenoviral vector of an alternate serotype. Hum Gene Ther (1997) 8(1):99-109.
18. Mastrangeli A, Harvey BG, Yao J, Wolff G, Kovesdi I, Crystal RG, Falck-Pedersen E: 'Sero-switch' adenovirus-mediated in vivo gene transfer: Circumvention of anti-adenovirus humoral immune defenses against repeat adenovirus vector administration by changing the adenovirus serotype. Hum Gene Ther (1996) 7(1):79-87.
19. Morral N, O'Neal W, Rice K, Leland M, Kaplan J, Piedra PA, Zhou H, Parks RJ, Velji R, Aguilar-Cordova E, Wadsworth S 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 USA (1999) 96(22):12816-12821.
20. Parks R, Evelegh C, Graham F: Use of helper-dependent adenoviral vectors of alternative serotypes permits repeat vector administration. Gene Ther (1999) 6(9):1565-1573.
21. Muruve DA, Barnes MJ, Stillman IE, Libermann TA: Adenoviral gene therapy leads to rapid induction of multiple chemokines and acute neutrophil-dependent hepatic injury in vivo. Hum Gene Ther (1999) 10(6):965-976.
22. 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(1):624-629.
23. Zhang Y, Chirmule N, Gao GP, Qian R, Croyle M, Joshi B, Tazelaar J, Wilson JM: Acute cytokine response to systemic adenoviral vectors in mice is mediated by dendritic cells and macrophages. Mol Ther (2001) 3(5 Pt 1):697-707.
24. Borgland SL, Bowen GP, Wong NC, Libermann TA, Muruve DA: Adenovirus vector-induced expression of the C-X-C chemokine IP-10 is mediated through capsid-dependent activation of NF-κB. J Virol (2000) 74(9):3941-3947.
25. Amalfitano A, Hauser MA, Hu H, Serra D, Begy CR, Chamberlain JS: Production and characterization of improved adenovirus vectors with the E1, E2b, and E3 genes deleted. J Virol (1998) 72(2):926-933.
26. Morral N, O'Neal W, Zhou H, Langston C, Beaudet A: Immune responses to reporter proteins and high viral dose limit duration of expression with adenoviral vectors: Comparison of E2a wild type and E2a deleted vectors. Hum Gene Ther (1997) 8(10):1275-1286.
27. 1- antitrypsin after systemic delivery. Hum Gene Ther (1998) 9(11):1587-1598.
28. Brann T, Kayda D, Lyons RM, Shirley P, Roy S, Kaleko M, Smith T: Adenoviral vector-mediated expression of physiologic levels of human factor VIII in nonhuman primates. Hum Gene Ther (1999) 10(18):2999-3011.
29. Christ M, Lusky M, Stoeckel F, Dreyer D, Dieterle A, Michou AI, Pavirani A, Mehtali M: Gene therapy with recombinant adenovinus vectors: Evaluation of the host immune response. Immunol Lett (1997) 57(1-3):19-25.
30. Hu H, Serra D, Amalfitano A: Persistence of an [E1-,polymerase-] adenovirus vector despite transduction of a neoantigen into immune-competent mice. Hum Gene Ther (1999) 10(3):355-364.
31. Ding EY, Hodges BL, McVie-Wylie AJ, Serra D, Pressley D, Chen YT, Amalfitano A: Long-term efficacy after [E1-,polymerase-] adenovirus-mediated transfer of human acid-α-glucosidase gene into glycogen storage disease type II knockout mice. Hum Gene Ther (2001) 12(8):955-965.
32. Harris JD, Graham IR, Schepelmann S, Stannard AK, Roberts ML, Hodges BL, Hill V, Amalfitano A, Hassall DG, Owen JS, Dickson G: Acute regression of advanced and retardation of early, aortic atheroma in immunocompetent apolipoprotein-E (apoE) deficient mice by administration of second generation [E1-,E3-,polymerase-] adenovirus vector expressing human apoE. Hum Mol Genet (2002) 11(1):43-58.
33. Luebke AE, Steiger JD, Hodges BL, Amalfitano A: A modified adenovirus can transfect cochlear hair cells in vivo without compromising cochlear function. Gene Ther (2001) 8(10):789-794.
34. Amalfitano A, Chamberlain JS: Isolation and characterization of packaging cell lines that coexpress the adenovirus E1, DNA polymerase, and preterminal proteins: Implications for gene therapy. Gene Ther (1997) 4(3):258-263
35. Langer SJ, Schaack J: 293 Cell lines that inducibly express high levels of adenovirus type 5 precursor terminal protein. Virology (1996) 221(1):172-179.
36. Moorhead JW, Clayton GH, Smith RL, Schaack J: A replication-incompetent adenovirus vector with the preterminal protein gene deleted efficiently transduces mouse ears. J Virol (1999) 73(2):1046-1053.
37. Hodges BL, Serra D, Hu H, Begy CA, Chamberlain JS, Amalfitano A: Multiply deleted [E1-, polymerase-, and pTP-] adenovirus vector persists despite deletion of the preterminal protein. J Gene Med (2000) 2(4):250-259.
38. Everett RE, Ding E, Serra D, Amalfitano A: Equivalent persistence of [E1-,E2b-] Ad vectors in diverse immunocompetent strains of mice accompanied by toxicity levels no different than those previously observed with use of 'gutted' Ad vectors. Fifth Annual Meeting of the American Society of Gene Therapy, Boston, MA, USA (2002).
39. Falgout B, Ketner G: Adenovirus early region 4 is required for efficient virus particle assembly. J Virol (1987) 61(12):3759-3768.
40. Lavoie JN, Nguyen M, Marcellus, RC, Branton PE, Shore GC: E4orf4, a novel -adenovirus death factor that induces p53-independent apoptosis by a pathway that is not inhibited by ZVAD-fmk. J Cell Biol (1998) 140(3):637-645.
41. Weinberg DH, Ketner G: Adenoviral early region 4 is required for efficient viral DNA replication and for late gene expression. J Virol (1986) 57(3):833-838.
42. Armentano D, Zabner J, Sacks C, Sookdeo CC, Smith MP, St George JA, Wadsworth SC, Smith AE, Gregory RJ: Effect of the E4 region on the persistence of transgene expression from adenovirus vectors. J Virol (1997) 71(3):2408-2416.
43. Dedieu JF, Vigne E, Torrent C, Jullien C, Mahfouz I, Caillaud JM, Aubailly N, Orsini C, Guillaume JM, Opolon P, Delaere P et al: Long-term gene delivery into the livers of immunocompetent mice with E1/E4-defective adenoviruses. J Virol (1997) 71(6):4626-4637.
44. Kaplan JM, Armentano D, Sparer TE, Wynn SG, Peterson PA, Wadsworth SC, Couture KK, Pennington SE, St George JA, Gooding LR, Smith AE: Characterization of factors involved in modulating persistence of transgene expression from recombinant adenovirus in the mouse lung. Hum Gene Ther (1997) B(1):45-56.
45. Wang Q, Greenburg G, Bunch D, Farson D, Finer MH: Persistent transgene expression in mouse liver following in vivo gene transfer with a δ-E1/δ-E4 adenovirus vector. Gene Ther (1997) 4(5):393-400.
46. Lusky M, Grave L, Dieterle A, Dreyer D, Christ M, Ziller C, Furstenberger P, Kintz J, Hadji DA, Pavirani A, Mehtali M: Regulation of adenovirus-mediated transgene expression by the viral E4 gene products: Requirement for E4 ORF3. J Virol (1999) 73(10):8308-8319.
47. Chirmule N, Hughes JV, Gao GP, Raper SE, Wilson JM: Role of E4 in eliciting CD4 T-cell and B-cell responses to adenovirus vectors delivered to murine and nonhuman primate lungs. J Virol (1998) 72(7):6138-6145.
48. Christ M, Louis B, Stoeckel F, Dieterle A, Grave L, Dreyer D, Kintz J, Ali Hadji D, Lusky M, Mehtali M: Modulation of the inflammatory properties and hepatotoxicity of recombinant adenovirus vectors by the viral E4 gene products. Hum Gene Ther (2000) 11(3):415-427.
49. 1-antitrypsin with negligible toxicity. Hum Gene Ther