Exploring gene-deleted adenoviral vectors for delivery of short hairpin RNAs and reduction of hepatitis B virus infection in mice

Journal of Gene Medicine

Volumn 10, Issue 8, 2008, Pages 878-889

  Rauschhuber, Christina ^a   Xu, Hui ^b   Salazar, Felix H ^c   Marion, Patricia L ^c   Ehrhardt, Anja ^a  

^a UNIVERSITY OF MUNICH   (Germany)

^b STANFORD UNIVERSITY SCHOOL OF MEDICINE   (United States)

^c STANFORD UNIVERSITY   (United States)

Author keywords

Adenovirus; Gene therapy; Hepatitis B virus; Mice; RNA interference; shRNA

Indexed keywords

ADENOVIRUS VECTOR; BETA GALACTOSIDASE; HEPATITIS B SURFACE ANTIBODY; LUCIFERASE; SHORT HAIRPIN RNA;

ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CONTROLLED STUDY; DNA SEQUENCE; GENE DELETION; GENE DELIVERY SYSTEM; GENE EXPRESSION; GENOMICS; HEPATITIS B; HEPATITIS B VIRUS; IN VITRO GENE TRANSFER; IN VIVO GENE TRANSFER; MOUSE; NONHUMAN; POSTTRANSCRIPTIONAL GENE SILENCING; PRIORITY JOURNAL; RNA INTERFERENCE; SOUTHERN BLOTTING; SUPERINFECTION; TRANSGENIC MOUSE; VIRUS CELL INTERACTION; VIRUS GENOME; VIRUS INHIBITION;

ADENOVIRIDAE; ANIMALIA; HEPATITIS B VIRUS; MUS; MUS MUSCULUS;

EID: 49349104504     PISSN: 1099498X     EISSN: 15212254     Source Type: Journal    
DOI: 10.1002/jgm.1207     Document Type: Article

References (36)

1. Rehermann B, Nascimbeni M. Immunology of hepatitis B virus and hepatitis C virus infection. Nat Rev Immunol 2005; 5: 215-229.
2. Wright TL. Introduction to chronic hepatitis B infection. Am J Gastroenterol 2006; 101: 1-6.
3. Zoulim F. Assessment of treatment efficacy in HBV infection and disease. J Hepatol 2006; 44: 95-99.
4. Tillmann HL. Antiviral therapy and resistance with hepatitis B virus infection. World J Gastroenterol 2007; 13: 125-40.
5. Wu J, Gerber MA. The inhibitory effects of antisense RNA on hepatitis B virus surface antigen synthesis. J Gen Virol 1997; 78: 641-647.
6. Madon J, Blum HE. Receptor-mediated delivery of hepatitis B virus DNA and antisense oligodeoxynucleotides to avian liver cells. Hepatology 1996; 24: 474-481.
7. Welch PJ, Tritz R, Yei S, et al. Intracellular application of hairpin ribozyme genes against hepatitis B virus. Gene Ther 1997; 4: 736-743.
8. Beck J, Nassal M. Efficient hammerhead ribozyme-mediated cleavage of the structured hepatitis B virus encapsidation signal in vitro and in cell extracts, but not in intact cells. Nucleic Acids Res 1995; 23: 4954-4962.
9. Scaglioni P, Melegari M, Takahashi M, et al. Use of dominant negative mutants of the hepadnaviral core protein as antiviral agents. Hepatology 1996; 24: 1010-1017.
10. Ogata N, Ostberg L, Ehrlich PH, et al. Markedly prolonged incubation period of hepatitis B in a chimpanzee passively immunized with a human monoclonal antibody to the a determinant of hepatitis B surface antigen. Proc Natl Acad Sci USA 1993; 90: 3014-3018.
11. McCaffrey AP, Nakai H, Pandey K, et al. Inhibition of hepatitis B virus in mice by RNA interference. Nat Biotechnol 2003; 21: 639-644.
12. 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.
13. Moore MD, McGarvey MJ, Russell RA, et al. Stable inhibition of hepatitis B virus proteins by small interfering RNA expressed from viral vectors. J Gene Med 2005; 7: 918-925.
14. Chen CC, Ko T-M, Ma H-I, et al. Long-term inhibition of hepatitis B virus in transgenic mice by double-stranded adeno-associated virus 8-delivered short hairpin RNA. Gene Ther 2007; 14: 11-19.
15. 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 USA 2005; 102: 773-778.
16. 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.
17. Gahery-Segard H, Farace F, Godfrin D, et al. Immune response to recombinant capsid proteins of adenovirus in humans: antifiber and anti-penton base antibodies have a synergistic effect on neutralizing activity. J Virol 1998; 72; 2388-2397.
18. Parks RJ, Chen L, Anton M, et al. A helper-dependent adenovirus vector system: removal of helper virus by Cre-mediated excision of the viral packaging signal. Proc Natl Acad Sci USA 1996; 93: 13565-13570.
19. Ehrhardt A, Kay MA. A new adenoviral helper-dependent vector results in long-term therapeutic levels of human coagulation factor IX at low doses in vivo. Blood 2002; 99: 3923-3930.
20. Cerullo V, Seiler MP, Mane V, et al. Correction of murine hemophilia A and immunological differences of factor VIII variants delivered by helper-dependent adenoviral vectors. Mol Ther 2007; 15: 2080-2087.
21. Oka K, Belalcazar LM, Dieker C, et al. Sustained phenotypic correction in a mouse model of hypoalphalipoproteinemia with a helper-dependent adenovirus vector. Gene Ther 2007; 14: 845.
22. Sano M, Kato Y, Taira K. Sequence-specific interference by small RNAs derived from adenovirus VAI RNA. FEBS Lett 2006; 580: 1553-1564.
23. McCaffrey AP, Meuse L, Pham T-TT, et al. RNA interference in adult mice. Nature 2002; 418: 38-39.
24. Ehrhardt A, Yant SR, Xu H, et al. Somatic integration from an adenoviral hybrid vector into a hot spot in mouse liver results in persistent transgene expression levels in vivo. Mol Ther 2007; 15: 146-156.
25. Ehrhardt A, Xu H, Dillow AM, et al. A gene-deleted adenoviral vector results in phenotypic correction of canine hemophilia B without liver toxicity or thrombocytopenia. Blood 2003; 102: 2403-2411.
26. Palmer D, Ng P. Improved system for helper-dependent adenoviral vector production. Mol Ther 2003; 8: 846-852.
27. 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.
28. Yang PL, Althage A, Chung J, Chisari FV. Hydrodynamic injection of viral DNA: a mouse model of acute hepatitis B virus infection. Proc Natl Acad Sci USA 2002; 99: 13825-13830.
29. Chen ZY, Yant SR, He C-Y, et al. Linear DNAs concatemerize in vivo and result in sustained transgene expression in mouse liver. Mol Ther 2001; 3: 403-410.
30. Marion PL, Salazar FH, Liittschwager K, et al. A transgenic mouse lineage useful for testing antivirals targeting hepatitis B virus. In Frontiers in Viral Hepatitis, Schinazi RF, ed.; Elsevier Science: Amsterdam, 2003; 197-209.
31. Nakai H, Fuess S, Storm TA, et al. Unrestricted hepatocyte transduction with adeno-associated virus serotype 8 vectors in mice. J Virol 2005; 79: 214-224.
32. Erdmann VA. Collection of published 5S and 5.8S RNA sequences and their precursors. Nucleic Acids Res 1979; 6: 29-44.
33. 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.
34. 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.
35. Cavanaugh VJ, Guidotti LG, Chisari FV. Inhibition of hepatitis B virus replication during adenovirus and cytomegalovirus infections in transgenic mice. J Virol 1998; 72: 2630-2637.
36. McClary H, Koch R, Chisari FV, et al. Relative sensitivity of hepatitis B virus and other hepatotropic viruses to the antiviral effects of cytokines. J Virol 2000; 74: 2255-2264.