Comparison of gene transfer to the murine liver following intraperitoneal and intraportal delivery of hepatotropic AAV pseudo-serotypes

Gene Therapy

Volumn 20, Issue 4, 2013, Pages 460-464

  Dane, A P ^a   Wowro, S J ^a   Cunningham, S C ^a,b   Alexander, I E ^a,b  

^a CHILDREN S HOSPITAL AT WESTMEAD   (Australia)

^b UNIVERSITY OF SYDNEY   (Australia)

Author keywords

AAV; capsid; expression pattern; liver; transduction; vector

Indexed keywords

ENHANCED GREEN FLUORESCENT PROTEIN; PARVOVIRUS VECTOR;

ADENO ASSOCIATED VIRUS; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; COMPARATIVE STUDY; FEMALE; GENE EXPRESSION; GENE TARGETING; GENE TRANSFER; MALE; NONHUMAN; PRIORITY JOURNAL; TRANSGENE;

ANIMALS; CAPSID; DEPENDOVIRUS; GENETIC VECTORS; INJECTIONS, INTRAPERITONEAL; INJECTIONS, INTRAVENOUS; LIVER; MICE; MICE, INBRED C57BL; PORTAL VEIN; TISSUE DISTRIBUTION; TRANSDUCTION, GENETIC; TRANSGENES;

ADENO-ASSOCIATED VIRUS; MIRIDAE; MURINAE;

EID: 84875917898     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2012.67     Document Type: Article

References (34)

1. Alexander IE, Cunningham SC, Logan GJ, Christodoulou J. Potential of AAV vectors in the treatment of metabolic disease. Gene Therapy 2008; 15: 831-839.
2. Jiang H, Couto LB, Patarroyo-White S, Liu T, Nagy D, Vargas JA et al. Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy. Blood 2006; 108: 3321-3328.
3. Nathwani AC, Rosales C, McIntosh J, Rastegarlari G, Nathwani D, Raj D et al. Longterm safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins. Mol Ther 2011; 19: 876-885.
4. Wang L, Bell P, Lin J, Calcedo R, Tarantal AF, Wilson JM. AAV8-mediated hepatic gene transfer in infant rhesus monkeys (Macaca mulatta). Mol Ther 2011; 19: 2012-2020.
5. Jiang H, Lillicrap D, Patarroyo-White S, Liu T, Qian X, Scallan CD et al. Multiyear therapeutic benefit of AAV serotypes 2, 6, and 8 delivering factor VIII to hemophilia A mice and dogs. Blood 2006; 108: 107-115.
6. Wang L, Calcedo R, Nichols TC, Bellinger DA, Dillow A, Verma IM et al. Sustained correction of disease in naive and AAV2-pretreated hemophilia B dogs: AAV2/8-mediated, liver-directed gene therapy. Blood 2005; 105: 3079-3086.
7. Nathwani AC, Tuddenham EG, Rangarajan S, Rosales C, McIntosh J, Linch DC et al. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N Engl J Med 2011; 365: 2357-2365.
8. Zaiss AK, Liu Q, Bowen GP, Wong NC, Bartlett JS, Muruve DA. Differential activation of innate immune responses by adenovirus and adeno-associated virus vectors. J Virol 2002; 76: 4580-4590.
9. Rabinowitz JE, Rolling F, Li C, Conrath H, Xiao W, Xiao X et al. Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity. J Virol 2002; 76: 791-801.
10. Thomas CE, Storm TA, Huang Z, Kay MA. Rapid uncoating of vector genomes is the key to efficient liver transduction with pseudotyped adeno-associated virus vectors. J Virol 2004; 78: 3110-3122.
11. Di Pasquale G, Chiorini JA. AAV transcytosis through barrier epithelia and endo-thelium. Mol Ther 2006; 13: 506-516.
12. Mitchell AM, Nicolson SC, Warischalk JK, Samulski RJ. AAV's anatomy: roadmap for optimizing vectors for translational success. Curr Gene Ther 2010; 10: 319-340.
13. Manno CS, Arruda VR, Pierce GF, Glader B, Ragni M, Rasko J et al. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nature Med 2006; 12: 342-347.
14. Mingozzi F, High KA. Immune responses to AAV in clinical trials. Curr Gene Ther 2007; 7: 316-324.
15. Sack BK, Herzog RW. Evading the immune response upon in vivo gene therapy with viral vectors. Curr Opin Mol Ther 2009; 11: 493-503.
16. Cunningham SC, Dane AP, Spinoulas A, Alexander IE. Gene delivery to the juvenile mouse liver using AAV2/8 Vectors. Mol Ther 2008; 16: 1081-1088.
17. Moscioni D, Morizono H, McCarter RJ, Stern A, Cabrera-Luque J, Hoang A et al. Long-term correction of ammonia metabolism and prolonged survival in orni-thine transcarbamylase-deficient mice following liver-directed treatment with novel adeno-associated viral vectors. Mol Ther 2006; 14: 25-33.
18. Dane AP, Cunningham SC, Graf NS, Alexander IE. Sexually dimorphic patterns of episomal rAAV genome persistence in the adult mouse liver and correlation with hepatocellular proliferation. Mol Ther 2009; 17: 1548-1554.
19. Bell P, Wang L, Gao G, Haskins ME, Tarantal AF, McCarter RJ et al. Inverse zonation of hepatocyte transduction with AAV vectors between mice and non-human primates. Mol Genet Metab 2011; 104: 395-403.
20. Jungermann K. Zonation of metabolism and gene expression in liver. Histochem Cell Biol 1995; 103: 81-91.
21. Davidoff AM, Ng CY, Zhou J, Spence Y, Nathwani AC. Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway. Blood 2003; 102: 480-488.
22. De BP, Heguy A, Hackett NR, Ferris B, Leopold PL, Lee J et al. High levels of persistent expression of alpha1-antitrypsin mediated by the nonhuman primate serotype rh.10 adeno-associated virus despite preexisting immunity to common human adeno-associated viruses. Mol Ther 2006; 13: 67-76.
23. Sun B, Zhang H, Franco LM, Brown T, Bird A, Schneider A et al. Correction of glycogen storage disease type II by an adeno-associated virus vector containing a muscle-specific promoter. Mol Ther 2005; 11: 889-898.
24. Lonning SM, Ford CA, Taylor KM, Hempel DM, Plog M, Ziegler RJ et al. Influence of gender-specific hormones on adeno-associated virus vector gene transfer and expression. Mol Ther 2002; 5: S2-S2.
25. Sun B, Zhang H, Franco LM, Young SP, Schneider A, Bird A et al. Efficacy of an adeno-associated virus 8-pseudotyped vector in glycogen storage disease type II. Mol Ther 2005; 11: 57-65.
26. Halbert DN, Cutt JR, Shenk T. Adenovirus early region 4 encodes functions required for efficient DNA replication, late gene expression, and host cell shutoff. J Virol 1985; 56: 250-257.
27. Ding W, Zhang L, Yan Z, Engelhardt JF. Intracellular trafficking of adeno-asso-ciated viral vectors. Gene Therapy 2005; 12: 873-880.
28. Wang J, Xie J, Lu H, Chen L, Hauck B, Samulski RJ et al. Existence of transient functional double-stranded DNA intermediates during recombinant AAV trans-duction. Proc Natl Acad Sci USA 2007; 104: 13104-13109.
29. Keiser NW, Yan Z, Zhang Y, Lei-Butters DC, Engelhardt JF. Unique characteristics of AAV1, 2, and 5 viral entry, intracellular trafficking, and nuclear import define transduction efficiency in HeLa cells. Hum Gene Ther 2011; 22: 1433-1444.
30. Nonnenmacher M, Weber T. Intracellular transport of recombinant adeno-asso-ciated virus vectors. Gene Therapy 2012; 19: 649-658.
31. Sonntag F, Bleker S, Leuchs B, Fischer R, Kleinschmidt JA. Adeno-associated virus type 2 capsids with externalized VP1/VP2 trafficking domains are generated prior to passage through the cytoplasm and are maintained until uncoating occurs in the nucleus. J Virol 2006; 80: 11040-11054.
32. Van Vliet K, Blouin V, Agbandje-McKenna M, Snyder RO. Proteolytic mapping of the adeno-associated virus capsid. Mol Ther 2006; 14: 809-821.
33. Lizee G, Aerts JL, Gonzales MI, Chinnasamy N, Morgan RA, Topalian SL. Real-time quantitative reverse transcriptase-polymerase chain reaction as a method for determining lentiviral vector titers and measuring transgene expression. Hum Gene Ther 2003; 14: 497-507.
34. Sambrook J, Fritsch EF, Maniatis T. Molecular Cloning a Laboratory Manual. Cold Spring Harbor Laboratory Press: Cold Spring Harbor, 1989.