Toxicity and adaptive immune response to intracellular transgenes delivered by helper-dependent vs. first generation adenoviral vectors

Molecular Genetics and Metabolism

Volumn 84, Issue 3 SPEC. ISS., 2005, Pages 278-288

  Mian, Asad ^a   Guenther, Margaretha ^a   Finegold, Milton ^a   Ng, Philip ^a   Rodgers, John ^a   Lee, Brendan ^a  

^a BAYLOR COLLEGE OF MEDICINE   (United States)

Author keywords

Adaptive immunity; Argininosuccinate synthetase; First generation adenovirus; Helper dependent adenovirus; Intracellular transgene; Urea cycle; Galactosidase

Indexed keywords

ADENOVIRUS VECTOR; BETA GALACTOSIDASE;

ARTICLE; CONTROLLED STUDY; CYTOARCHITECTURE; GENE EXPRESSION; GENE VECTOR; GENOME; IMMUNE DEFICIENCY; IMMUNE RESPONSE; IMMUNOGENICITY; IMMUNOTOXICITY; LIVER CELL; MOUSE; NONHUMAN; PRIORITY JOURNAL; PROTEIN EXPRESSION; SIGNAL TRANSDUCTION; TRANSGENE; WILD TYPE;

EID: 13444280019     PISSN: 10967192     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.ymgme.2004.11.005     Document Type: Article

References (39)

1. A. Amalfitano, and R.J. Parks Separating fact from fiction: assessing the potential of modified adenovirus vectors for use in human gene therapy Curr. Gene Ther. 2 2 2002 111 133
2. J. Roy-Chowdhury, and M.S. Horwitz Evolution of adenoviruses as gene therapy vectors Mol. Ther. 5 4 2002 340 344
3. A. Mastrangeli "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. 7 1 1996 79 87
4. C.A. Mack Circumvention of anti-adenovirus neutralizing immunity by administration of an adenoviral vector of an alternate serotype Hum. Gene Ther. 8 1 1997 99 109
5. G.P. Gao, Y. Yang, and J.M. Wilson Biology of adenovirus vectors with E1 and E4 deletions for liver-directed gene therapy J. Virol. 70 12 1996 8934 8943
6. M. Brenner Gene transfer by adenovectors Blood 94 12 1999 3965 3967
7. M. Brenner Reports of adenovector "death" are greatly exaggerated Mol. Ther. 1 3 2000 205
8. Q. Liu The role of capsid-endothelial interactions in the innate immune response to adenovirus vectors Hum. Gene Ther. 14 7 2003 627 643
9. K. Jooss, H.C. Ertl, and J.M. Wilson Cytotoxic T-lymphocyte target proteins and their major histocompatibility complex class I restriction in response to adenovirus vectors delivered to mouse liver J. Virol. 72 4 1998 2945 2954
10. Y. Yang Immune responses to viral antigens versus transgene product in the elimination of recombinant adenovirus-infected hepatocytes in vivo Gene Ther. 3 2 1996 137 144
11. K. Oka Long-term stable correction of low-density lipoprotein receptor-deficient mice with a helper-dependent adenoviral vector expressing the very low-density lipoprotein receptor Circulation 103 9 2001 1274 1281
12. I.H. Kim Lifetime correction of genetic deficiency in mice with a single injection of helper-dependent adenoviral vector Proc. Natl. Acad. Sci. USA 98 23 2001 13282 13287
13. B. Lee Hepatocyte gene therapy in a large animal: a neonatal bovine model of citrullinemia Proc. Natl. Acad. Sci. USA 96 7 1999 3981 3986
14. X. Ye Correction of argininosuccinate synthetase (AS) deficiency in a murine model of citrullinemia with recombinant adenovirus carrying human AS cDNA Gene Ther. 7 20 2000 1777 1782
15. K.N. Wills Development and characterization of recombinant adenoviruses encoding human p53 for gene therapy of cancer Hum. Gene Ther. 5 9 1994 1079 1088
16. R.J. Parks A helper-dependent adenovirus vector system: removal of helper virus by Cre-mediated excision of the viral packaging signal Proc. Natl. Acad. Sci. USA 93 24 1996 13565 13570
17. P. Ng, R.J. Parks, and F.L. Graham Preparation of helper-dependent adenoviral vectors Methods Mol. Med. 69 2002 371 388
18. G. Toietta Generation of helper-dependent adenoviral vectors by homologous recombination Mol. Ther. 5 2 2002 204 210
19. A.F. Ochsenbein Roles of tumour localization, second signals and cross priming in cytotoxic T-cell induction Nature 411 6841 2001 1058 1064
20. S.K. Tripathy Stable delivery of physiologic levels of recombinant erythropoietin to the systemic circulation by intramuscular injection of replication-defective adenovirus Proc. Natl. Acad. Sci. USA 91 24 1994 11557 11561
21. N. Morral 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. 8 10 1997 1275 1286
22. M.A. Kay Therapeutic serum concentrations of human alpha-1-antitrypsin after adenoviral-mediated gene transfer into mouse hepatocytes Hepatology 21 3 1995 815 819
23. S. Connelly In vivo gene delivery and expression of physiological levels of functional human factor VIII in mice Hum. Gene Ther. 6 2 1995 185 193
24. M.A. Kay In vivo hepatic gene therapy: complete albeit transient correction of factor IX deficiency in hemophilia B dogs Proc. Natl. Acad. Sci. USA 91 6 1994 2353 2357
25. T. Ito Rapid screening of high-risk patients for disorders of purine and pyrimidine metabolism using HPLC-electrospray tandem mass spectrometry of liquid urine or urine-soaked filter paper strips Clin. Chem. 46 4 2000 445 452
26. G. Schiedner Genomic DNA transfer with a high-capacity adenovirus vector results in improved in vivo gene expression and decreased toxicity Nat. Genet. 18 2 1998 180 183
27. D.A. Muruve Adenoviral gene therapy leads to rapid induction of multiple chemokines and acute neutrophil-dependent hepatic injury in vivo Hum. Gene Ther. 10 6 1999 965 976
28. L. Pastore Use of a liver-specific promoter reduces immune response to the transgene in adenoviral vectors Hum. Gene Ther. 10 11 1999 1773 1781
29. N. Morral 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 96 22 1999 12816 12821
30. N. Morral High doses of a helper-dependent adenoviral vector yield supraphysiological levels of alpha1-antitrypsin with negligible toxicity Hum. Gene Ther. 9 18 1998 2709 2716
31. M.A. Morsy An adenoviral vector deleted for all viral coding sequences results in enhanced safety and extended expression of a leptin transgene Proc. Natl. Acad. Sci. USA 95 14 1998 7866 7871
32. C. Balague Sustained high-level expression of full-length human factor VIII and restoration of clotting activity in hemophilic mice using a minimal adenovirus vector Blood 95 3 2000 820 828
33. P.S. Reddy Sustained human factor VIII expression in hemophilia A mice following systemic delivery of a gutless adenoviral vector Mol. Ther. 5 1 2002 63 73
34. A. Ehrhardt A gene-deleted adenoviral vector results in phenotypic correction of canine hemophilia B without liver toxicity or thrombocytopenia Blood 102 7 2003 2403 2411
35. H.H. Chen Persistence in muscle of an adenoviral vector that lacks all viral genes Proc. Natl. Acad. Sci. USA 94 5 1997 1645 1650
36. A. Mian Long-term correction of ornithine transcarbamylase deficiency by WPRE-mediated overexpression using a helper-dependent adenovirus Mol. Ther. 10 3 2004 492 499
37. R. Gilbert Dystrophin expression in muscle following gene transfer with a fully deleted (gutted) adenovirus is markedly improved by trans-acting adenoviral gene products Hum. Gene Ther. 12 14 2001 1741 1755
38. R. Gilbert Improved performance of a fully gutted adenovirus vector containing two full-length dystrophin cDNAs regulated by a strong promoter Mol. Ther. 6 4 2002 501 509
39. M.D. Roth Helper-dependent adenoviral vectors efficiently express transgenes in human dendritic cells but still stimulate antiviral immune responses J. Immunol. 169 8 2002 4651 4656