AAV5-mediated gene transfer to the parotid glands of non-human primates

Gene Therapy

Volumn 17, Issue 1, 2010, Pages 50-60

  Voutetakis, A ^a,d   Zheng, C ^a   Cotrim, A P ^a   Mineshiba, F ^a,e   Afione, S ^a   Roescher, N ^a   Swaim, W D ^a   Metzger, M ^b   Eckhaus, M A ^c   Donahue, R E ^b   Dunbar, C E ^b   Chiorini, J A ^a   Baum, B J ^a  

^a NATIONAL INSTITUTE OF DENTAL AND CRANIOFACIAL RESEARCH   (United States)

^b NATIONAL HEART LUNG AND BLOOD INSTITUTE   (United States)

^c NATIONAL INSTITUTES OF HEALTH   (United States)

^d UNIVERSITY OF ATHENS   (Greece)

^e OKAYAMA UNIVERSITY   (Japan)

Author keywords

AAV; Gene therapeutics; Non human primates; Salivary glands; Serotype 5

Indexed keywords

ERYTHROPOIETIN; PARVOVIRUS VECTOR;

ADENOVIRUS 2; ADENOVIRUS 5; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; DNA STRUCTURE; DNA VECTOR; GENE EXPRESSION; GENE TARGETING; MACACA; MALE; MOLECULAR MECHANICS; NONHUMAN; PAROTID GLAND; POLYMERASE CHAIN REACTION; PRIORITY JOURNAL; STRAIN DIFFERENCE; VIRAL GENE DELIVERY SYSTEM; VIRUS STRAIN;

ANIMALS; DEPENDOVIRUS; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; MACACA MULATTA; PAROTID GLAND; TRANSDUCTION, GENETIC; TRANSGENES;

MACACA; MACACA MULATTA; MURINAE; PRIMATES;

EID: 74349121109     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2009.123     Document Type: Article

References (32)

1. Zufferey R, Aebischer P. Salivary glands and gene therapy: the mouth waters. Gene Therapy 2004; 11: 1425-1426.
2. Baum BJ, Voutetakis A, Wang J. Salivary glands: novel target sites for gene therapeutics. Trends Mol Med 2004; 10: 585-590.
3. Shan Z, Li J, Zheng C, Liu X, Zhang C, Goldsmith CM et al. Increased fluid secretion after adenoviral-mediated transfer of the human aquaporin-1 cDNA to irradiated miniature pig parotid glands. Mol Ther 2005; 11: 444-451.
4. Wang J, Voutetakis A, Zheng C, Baum BJ. Rapamycin control of exocrine protein levels in saliva after adenoviral vector-mediated gene transfer. Gene Therapy 2004; 11: 729-733.
5. Voutetakis A, Kok MR, Zheng C, Bossis I, Wang J, Cotrim AP et al. Reengineered salivary glands are stable endogenous bioreactors for systemic gene therapeutics. Proc Natl Acad Sci USA 2004; 101: 3053-3058.
6. Ferber D. Gene therapy: safer and virus free? Science 2001; 294: 1638-1642.
7. Casals M, Haskins M. Large animal models and gene therapy. Eur J Hum Genet 2006; 14: 266-272.
8. Voutetakis A, Zheng C, Mineshiba F, Cotrim AP, Goldsmith CM, Schmidt M et al. Adeno-associated virus serotype 2-mediated gene transfer to the parotid glands of nonhuman primates. Hum Gene Ther 2007; 18: 142-150.
9. Katano H, Kok MR, Cotrim AP, Yamano S, Schmidt M, Afione S et al. Enhanced transduction of mouse salivary glands with AAV5-based vectors. Gene Therapy 2006; 13: 594-601.
10. Di Pasquale G, Davidson BL, Stein CS, Martins I, Scudiero D, Monks A et al. Identification of PDGFR as a receptor for AAV-5 transduction. Nat Med 2003; 9: 849-855.
11. Kampf D, Eckardt KU, Fischer HC, Schmalisch CB, Schostak M. Pharmacokinetics of recombinant erythropoietin in dialysis patients after single and multiple subcutaneous administrations. Nephron 1992; 61: 393-398.
12. Duan D, Sharma P, Yang J, Yue Y, Dudus L, Zhang Y, et al. Circular intermediates of recombinant adeno-associated virus have defined structural characteristics responsible for long-term episomal persistence in muscle tissue. J Virol 1998; 73: 8568-8577.
13. Song S, Laipis PJ, Berns KI, Flotte TR. Effect of DNA-dependent protein kinase on the molecular fate of the rAAV2 genome in skeletal muscle. Proc Natl Acad Sci USA 2001; 98: 4084-4088.
14. Yan Z, Zak R, Zhang Y, Engelhardt JF. Inverted terminal repeat sequences are important for intermolecular recombination and circularization of adeno-associated virus genomes. J Virol 2005; 79: 364-379.
15. Manno CS, Pierce GF, Arruda VR, Glader B, Ragni M, Rasko JJE et al. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med 2006; 12: 342-347.
16. Mingozzi F, Maus MV, Hui DJ, Sabatino DE, Murphy SL, Rasko JE et al. CD8+ T-cell responses to adeno-associated virus capsid in humans. Nat Med 2007; 13: 419-422.
17. Kok MR, Yamano S, Lodde BM, Wang J, Couwenhoven RI, Yakar S et al. Local adeno-associated virus-mediated interleukin 10 gene transfer has disease-modifying effects in a murine model of Sjogren's syndrome. Hum Gene Ther 2003; 14: 1605-1618.
18. Nathwani AC, Gray JT, McIntosh J, Ng CYC, Zhou J, Spence Y et al. Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human Factor IX in non-human primates. Blood 2007; 109: 1414-1421.
19. Auricchio A, O'Connor E, Weiner D, Gao GP, Hildinger M, Wang L et al. Noninvasive gene transfer to the lung for systemic delivery of therapeutic proteins. J Clin Invest 2002; 110: 499-504.
20. Chiorini JA, Kim F, Yang L, Kotin RM. Cloning and characterization of adeno-associated virus type 5. J Virol 1999; 73: 1309-1319.
21. Chiorini JA, Afione S, Kotin RM. Adeno-associated virus (AAV) type 5 Rep protein cleaves a unique terminal resolution site compared with other AAV serotypes. J Virol 1999; 73: 4293-4298.
22. Schmidt M, Katano H, Bossis I, Chiorini JA. Cloning and characterization of a bovine adeno-associated virus. J Virol 2004; 78: 6509-6516.
23. Yan Z, Lei-Butters DC, Zhang Y, Zak R, Engelhardt JF. Hybrid adeno-associated virus bearing nonhomologous inverted terminal repeats enhances dual-vector reconstruction of minigenes in vivo. Hum Gene Ther 2007; 18: 81-87.
24. Brister JR, Muzyczka N. Rep-mediated nicking of the adeno-associated virus origin requires two biochemical activities, DNA helicase activity and transesterification. J Virol 1999; 73: 9325-9336.
25. Lotery AJ, Yang GS, Mullins RF, Russell SR, Schmidt M, Stone EM et al. Adeno-associated virus type 5: transduction efficiency and cell-type specificity in the primate retina. Hum Gene Ther 2003; 14: 1663-1671.
26. Scallan CD, Jiang H, Liu T, Patarroyo-White S, Sommer JM, Zhou S et al. Human immunoglobulin inhibits liver transduction by AAV vectors at low AAV2 neutralizing titers in SCID mice. Blood 2006; 107: 1810-1817.
27. Summerford C, Samulski RJ. Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions. J Virol 1998; 72: 1438-1445.
28. Kaludov N, Handelman B, Chiorini JA. Scalable purification of adeno-associated virus type 2, 4, or 5 using ion-exchange chromatography. Hum Gene Ther 2002; 13: 1235-1243.
29. Lee GM, Thornwaite JT, Rasch EM. Picogram per cell determination of DNA by flow cytofluorometry. Analyt Biochem 1984; 137: 221-226.
30. Rivera VM, Gao GP, Grant RL, Schnell MA, Zoltick PW, Rozamus LW et al. Long-term pharmacologically regulated expression of erythropoietin in primates following AAV-mediated gene transfer. Blood 2005; 105: 1424-1430.
31. Voutetakis A, Zheng C, Metzger M, Cotrim AP, Donahue RE, Dunbar CE et al. Sorting of transgenic secretory proteins in rhesus macaque parotid glands following adenoviral mediated gene transfer. Hum Gene Ther 2008; 19: 1401-1405.
32. Zheng C, Vitolo JM, Zhang W, Mineshiba F, Chiorini JA, Baum BJ. Extended transgene expression from a nonintegrating adenoviral vector containing retroviral elements. Mol Ther 2008; 16: 1089-1097.