Inhibition of HBV replication in vivo using helper-dependent adenovirus vectors to deliver antiviral RNA interference expression cassettes

Antiviral Therapy

Volumn 19, Issue 4, 2014, Pages 363-373

  Crowther, Carol ^a   Mowa, Mohube B ^a   Ely, Abdullah ^a   Arbuthnot, Patrick B ^a  

^a UNIVERSITY OF THE WITWATERSRAND   (South Africa)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; HEPATITIS B ANTIBODY; SHORT HAIRPIN RNA; CYTOKINE; SMALL INTERFERING RNA; VIRUS RNA;

ADAPTIVE IMMUNITY; ADENOVIRUS; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CYTOKINE RELEASE; GENE EXPRESSION; GENE SILENCING; GENETIC TRANSDUCTION; HEPATITIS B; HEPATITIS B VIRUS; HUMAN; HUMAN CELL; HUMORAL IMMUNITY; IMMUNE RESPONSE; IMMUNOSTIMULATION; INNATE IMMUNITY; LACZ GENE; LIVER CELL; LIVER TOXICITY; MOUSE; NONHUMAN; PRIORITY JOURNAL; REPORTER GENE; RNA INTERFERENCE; RNA PROCESSING; TRANSGENE; VIRAL GENE DELIVERY SYSTEM; VIRUS GENOME; VIRUS LOAD; VIRUS PARTICLE; VIRUS REPLICATION; ADENOVIRIDAE; ANIMAL; BLOOD; CELL LINE; GENE ORDER; GENE TRANSFER; GENE VECTOR; GENETICS; HELPER VIRUS; PHYSIOLOGY; TRANSGENIC MOUSE;

ADENOVIRIDAE; ANIMALS; CELL LINE; CYTOKINES; GENE EXPRESSION; GENE ORDER; GENE TRANSFER TECHNIQUES; GENES, REPORTER; GENETIC VECTORS; GENOME, VIRAL; HELPER VIRUSES; HEPATITIS B VIRUS; HUMANS; MICE, TRANSGENIC; RNA INTERFERENCE; RNA, SMALL INTERFERING; RNA, VIRAL; TRANSDUCTION, GENETIC; VIRUS REPLICATION;

EID: 84922433927     PISSN: 13596535     EISSN: 20402058     Source Type: Journal    
DOI: 10.3851/IMP2713     Document Type: Article

References (50)

1. Organziation WH. Hepatitis B. Factsheet number 204 (Updated July 2013. Accessed 11 June 2014.) Available from http://www.who.int/mediacentre/ factsheets/fs204/en/index.html
2. Arbuthnot P, Kew M. Hepatitis B virus and hepatocellular carcinoma. Int J Exp Pathol 2001; 82:77-100.
3. Keeffe EB, Dieterich DT, Han SH, et al. A treatment algorithm for the management of chronic hepatitis B virus infection in the United States: an update. Clin Gastroenterol Hepatol 2006; 4:936-962.
4. Keeffe EB, Dieterich DT, Pawlotsky JM, Benhamou Y. Chronic hepatitis B: preventing, detecting, and managing viral resistance. Clin Gastroenterol Hepatol 2008; 6:268-274.
5. Arbuthnot P. Harnessing RNA interference for the treatment of viral infections. Drug News Perspect 2010; 23:341-350.
6. Chen Y, Cheng G, Mahato RI. RNAi for treating hepatitis B viral infection. Pharm Res 2008; 25:72-86.
7. Ivacik D, Ely A, Arbuthnot P. Countering hepatitis B virus infection using RNAi: how far are we from the clinic? Rev Med Virol 2011; 21:383-396.
8. McCaffrey AP. RNA interference inhibitors of hepatitis B virus. Ann N Y Acad Sci 2009; 1175:15-23.
9. Castanotto D, Rossi JJ. The promises and pitfalls of RNA-interference- based therapeutics. Nature 2009; 457:426-433.
10. Brummelkamp TR, Bernards R, Agami R. A system for stable expression of short interfering RNAs in mammalian cells. Science 2002; 296:550-553.
11. Lee NS, Dohjima T, Bauer G, et al. Expression of small interfering RNAs targeted against HIV-1 rev transcripts in human cells. Nat Biotechnol 2002; 20:500-505.
12. Miyagishi M, Taira K. U6 promoter driven siRNAs with four uridine 3′ overhangs efficiently suppress targeted gene expression in mammalian cells. Nat Biotechnol 2002; 20:497-500.
13. Paul CP, Good PD, Winer I, Engelke DR. Effective expression of small interfering RNA in human cells. Nat Biotechnol 2002; 20:505-508.
14. Mowa MB, Crowther C, Arbuthnot P. Therapeutic potential of adenoviral vectors for delivery of expressed RNAi activators. Expert Opin Drug Deliv 2010; 7:1373-1385.
15. 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.
16. Taniguchi M, Seino K, Nakayama T. The NKT cell system: bridging innate and acquired immunity. Nat Immunol 2003; 4:1164-1165.
17. Chen Q, Wei H, Sun R, Zhang J, Tian Z. Therapeutic RNA silencing of Cys-X3-Cys chemokine ligand 1 gene prevents mice from adenovirus vector-induced acute liver injury. Hepatology 2008; 47:648-658.
18. Guidotti LG, Chisari FV. Noncytolytic control of viral infections by the innate and adaptive immune response. Annu Rev Immunol 2001; 19:65-91.
19. Carmona S, Ely A, Crowther C, et al. Effective inhibition of HBV replication in vivo by anti-HBx short hairpin RNAs. Mol Ther 2006; 13:411-421.
20. Crowther C, Ely A, Hornby J, et al. Efficient inhibition of hepatitis B virus replication in vivo, using polyethylene glycol-modified adenovirus vectors. Hum Gene Ther 2008; 19:1325-1331.
21. Uprichard SL, Boyd B, Althage A, Chisari FV. Clearance of hepatitis B virus from the liver of transgenic mice by short hairpin RNAs. Proc Natl Acad Sci U S A 2005; 102:773-778.
22. Mowa MB, Crowther C, Ely A, Arbuthnot P. Efficient silencing of hepatitis B virus by helper-dependent adenovirus vector-mediated delivery of antiviral primary micro RNA shuttles. Micro RNA 2012; 1:19-25.
23. McCoy RD, Davidson BL, Roessler BJ, et al. Pulmonary inflammation induced by incomplete or inactivated adenoviral particles. Hum Gene Ther 1995; 6:1553-1560.
24. Raper SE, Yudkoff M, Chirmule N, et al. A pilot study of in vivo liver-directed gene transfer with an adenoviral vector in partial ornithine transcarbamylase deficiency. Hum Gene Ther 2002; 13:163-175.
25. Yei S, Mittereder N, Wert S, Whitsett JA, Wilmott RW, Trapnell BC. In vivo evaluation of the safety of adenovirus-mediated transfer of the human cystic fibrosis transmembrane conductance regulator cDNA to the lung. Hum Gene Ther 1994; 5:731-744.
26. Rauschhuber C, Xu H, Salazar FH, Marion PL, Ehrhardt A. Exploring gene-deleted adenoviral vectors for delivery of short hairpin RNAs and reduction of hepatitis B virus infection in mice. J Gene Med 2008; 10:878-889.
27. Marion PL, Salazar FH, Liittschwager K, et al. A transgenic mouse lineage useful for testing antivirals targeting hepatitis B virus. In Schinazi RF, Sommadossi J-P, Rich CM (Editors). Frontiers in viral hepatitis. Amsterdam: Elsevier Science 2003; pp.197-202.
28. He TC, Zhou S, da Costa LT, Yu J, Kinzler KW, Vogelstein B. A simplified system for generating recombinant adenoviruses. Proc Natl Acad Sci U S A 1998; 95:2509-2514.
29. Palmer D, Ng P. Improved system for helper-dependent adenoviral vector production. Mol Ther 2003; 8:846-852.
30. Mok H, Palmer DJ, Ng P, Barry MA. Evaluation of polyethylene glycol modification of first-generation and helper-dependent adenoviral vectors to reduce innate immune responses. Mol Ther 2005; 11:66-79.
31. Weinberg MS, Ely A, Barichievy S, et al. Specific inhibition of HBV replication in vitro and in vivo with expressed long hairpin RNA. Mol Ther 2007; 15:534-541.
32. Jiang H, Wang Z, Serra D, Frank MM, Amalfitano A. Recombinant adenovirus vectors activate the alternative complement pathway, leading to the binding of human complement protein C3 independent of anti-ad antibodies. Mol Ther 2004; 10:1140-1142.
33. He L, Hakimi J, Salha D, Miron I, Dunn P, Radvanyi L. A sensitive flow cytometry-based cytotoxic T-lymphocyte assay through detection of cleaved caspase 3 in target cells. J Immunol Methods 2005; 304:43-59.
34. Ely A, Naidoo T, Mufamadi S, Crowther C, Arbuthnot P. Expressed anti-HBV primary microRNA shuttles inhibit viral replication efficiently in vitro and in vivo. Mol Ther 2008; 16:1105-1112.
35. Jooss K, Yang Y, Fisher KJ, Wilson JM. Transduction of dendritic cells by DNA viral vectors directs the immune response to transgene products in muscle fibers. J Virol 1998; 72:4212-4223.
36. Brunetti-Pierri N, Clarke C, Mane V, et al. Phenotypic correction of ornithine transcarbamylase deficiency using low dose helper-dependent adenoviral vectors. J Gene Med 2008; 10:890-896.
37. Brunetti-Pierri N, Stapleton GE, Law M, et al. Efficient, long-term hepatic gene transfer using clinically relevant HDAd doses by balloon occlusion catheter delivery in nonhuman primates. Mol Ther 2009; 17:327-333.
38. Zaiss AK, Machado HB, Herschman HR. The influence of innate and pre-existing immunity on adenovirus therapy. J Cell Biochem 2009; 108:778-790.
39. Cotter MJ, Muruve DA. The induction of inflammation by adenovirus vectors used for gene therapy. Front Biosci 2005; 10:1098-1105.
40. Liu Q, Zaiss AK, Colarusso P, et al. The role of capsid-endothelial interactions in the innate immune response to adenovirus vectors. Hum Gene Ther 2003; 14:627-643.
41. McCaffrey AP, Fawcett P, Nakai H, et al. The host response to adenovirus, helper-dependent adenovirus, and adeno-associated virus in mouse liver. Mol Ther 2008; 16:931-941.
42. Muruve DA. The innate immune response to adenovirus vectors. Hum Gene Ther 2004; 15:1157-1166.
43. Muruve DA, Cotter MJ, Zaiss AK, et al. Helper-dependent adenovirus vectors elicit intact innate but attenuated adaptive host immune responses in vivo. J Virol 2004; 78:5966-5972.
44. Arbuthnot P, Longshaw V, Naidoo T, Weinberg MS. Opportunities for treating chronic hepatitis B and C virus infection using RNA interference. J Viral Hepat 2007; 14:447-459.
45. Brunetti-Pierri N, Ng P. Helper-dependent adenoviral vectors for liver-directed gene therapy. Hum Mol Genet 2011; 20:R7-R13.
46. Brunetti-Pierri N, Ng T, Iannitti DA, et al. Improved hepatic transduction, reduced systemic vector dissemination, and long-term transgene expression by delivering helper-dependent adenoviral vectors into the surgically isolated liver of nonhuman primates. Hum Gene Ther 2006; 17:391-404.
47. Grimm D, Streetz KL, Jopling CL, et al. Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways. Nature 2006; 441:537-541.
48. Danielsson A, Elgue G, Nilsson BM, et al. An ex vivo loop system models the toxicity and efficacy of PEGylated and unmodified adenovirus serotype 5 in whole human blood. Gene Ther 2010; 17:752-762.
49. Schiffer JT, Aubert M, Weber ND, Mintzer E, Stone D, Jerome KR. Targeted DNA mutagenesis for the cure of chronic viral infections. J Virol 2012; 86:8920-8936.
50. Moolla N, Kew M, Arbuthnot P. Regulatory elements of hepatitis B virus transcription. J Viral Hepat 2002; 9:323-331.