Comparative efficacy of intratracheal adeno-associated virus administration to newborn rats

Human Gene Therapy

Volumn 16, Issue 11, 2005, Pages 1298-1306

  Fleurence, Emmanuelle ^a   Riviere, Christel ^b   Lacaze Masmonteil, Thierry ^c   Franco Motoya, Marie Laure ^a   Waszak, Paul ^a   Bourbon, Jacques ^a   Danos, Olivier ^b   Douar, Anne Marie ^b   Delacourt, Christophe ^a,d  

^a INSERM   (France)

^b GÉNÉTHON   (France)

^c HÔPITAL ANTOINE BÉCLÈRE   (France)

^d CENTRE HOSPITALIER INTERCOMMUNAL DE CRÉTEIL   (France)

Author keywords

[No Author keywords available]

Indexed keywords

BETA GALACTOSIDASE; LUCIFERASE; PARVOVIRUS VECTOR; RECOMBINANT PROTEIN;

ADENO ASSOCIATED VIRUS; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BODY WEIGHT; CELL COUNT; CELL IMMORTALIZATION; CONTROLLED STUDY; DOSE RESPONSE; FETUS LUNG; GENE OVEREXPRESSION; GENETIC TRANSDUCTION; HUMAN; HUMAN CELL; IN VITRO STUDY; INFLAMMATORY CELL; LUNG ALVEOLUS EPITHELIUM; LUNG ALVEOLUS MACROPHAGE; LUNG DEVELOPMENT; LUNG DYSPLASIA; LUNG LAVAGE; LUNG STRUCTURE; LUNG VOLUME; MESENCHYME CELL; MORPHOMETRICS; NEWBORN; NONHUMAN; PREMATURITY; RAT; RECOMBINANT ADENO ASSOCIATED VIRUS 1; RECOMBINANT ADENO ASSOCIATED VIRUS 2; RECOMBINANT ADENO ASSOCIATED VIRUS 5; TRANSGENE; VIRAL GENE THERAPY; VIRUS RECOMBINANT;

ADENO-ASSOCIATED VIRUS; ADENO-ASSOCIATED VIRUS 1; ADENO-ASSOCIATED VIRUS 2; ADENO-ASSOCIATED VIRUS 5; ANIMALIA; PARVOVIRUS;

EID: 27744511760     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/hum.2005.16.1298     Document Type: Article

References (56)

1. ALLEN, J.M., HALBERT, C.L., and MILLER, A.D. (2000). Improved adeno-associated virus vector production with transfection of a single helper adenovirus gene, E4orf6. Mol. Ther. 1, 88-95.
2. AURICCHIO, A., O'CONNOR, E., WEINER, D., GAO, G.P., HILDINGER, M., WANG, L., CALCEDO, R., and WILSON, J.M. (2002). Noninvasive gene transfer to the lung for systemic delivery of therapeutic proteins. J. Clin. Invest. 110, 499-504.
3. BALS, R., XIAO, W., SANG, N., WEINER, D.J., MEEGALLA, R.L., and WILSON, J.M. (1999). Transduction of well-differentiated airway epithelium by recombinant adeno-associated virus is limited by vector entry. J. Virol. 73, 6085-6088.
4. BURRI, P.H., DBALY, J., and WEIBEL, E.R. (1974). The postnatal growth of the rat lung. I. Morphometry. Anat. Rec. 178, 711-730.
5. CHIORINI, J.A., YANG, L., LIU, Y., SAFER, B., and KOTIN, R.M. (1997). Cloning of adeno-associated virus type 4 (AAV4) and generation of recombinant AAV4 particles. J. Virol. 71, 6823-6833.
6. CHIORINI, J.A., AFIONE, S., and KOTIN, R.M. (1999a). Adeno-associated virus (AAV) type 5 Rep protein cleaves a unique terminal resolution site compared with other AAV serotypes. J. Virol. 73, 4293-4298.
7. CHIORINI, J.A., KIM, F., YANG, L., and KOTIN, R.M. (1999b). Cloning and characterization of adeno-associated virus type 5. J. Virol. 73, 1309-1319.
8. DALY, T.M. (2004). Overview of adeno-associated viral vectors. Methods Mol. Biol. 246, 157-165.
9. DAVIDSON, B.L., STEIN, C.S., HETH, J.A., MARTINS, I., KOTIN, R.M., DERKSEN, T.A., ZABNER, J., GHODSI, A., and CHIORINI, J.A. (2000). Recombinant adeno-associated virus type 2, 4, and 5 vectors: Transduction of variant cell types and regions in the mammalian central nervous system. Proc. Natl. Acad. Sci. U.S.A. 97, 3428-3432.
10. DELACOURT, C., D'ORTHO, M.P., MACQUIN-MAVIER, I., PEZET, S., HARF, A., and LAFUMA, C. (1995). Increased 92 kD gelatinase activity from alveolar macrophages in newborn rats. Am. J. Respir. Crit. Care Med. 151, 1939-1945.
11. DING, W., YAN, Z., ZAK, R., SAAVEDRA, M., RODMAN, D.M., and ENGELHARDT, J.F. (2003). Second-strand genome conversion of adeno-associated virus type 2 (AAV-2) and AAV-5 is not rate limiting following apical infection of polarized human airway epithelia. J. Virol. 77, 7361-7366.
12. DI PASQUALE, G., DAVIDSON, B.L., STEIN, C.S., MARTINS, I., SCUDIERO, D., MONKS, A., and CHIORINI, J.A. (2003). Identification of PDGFR as a receptor for AAV-5 transduction. Nat. Med. 9, 1306-1312.
13. DOUAR, A.M., POULARD, K., STOCKHOLM, D., and DANOS, O. (2001). Intracellular trafficking of adeno-associated virus vectors: Routing to the late endosomal compartment and proteasome degradation. J. Virol. 75, 1824-1833.
14. DRISKELL, R.A., and ENGELHARDT, J.F. (2003). Current status of gene therapy for inherited lung diseases. Annu. Rev. Physiol. 65, 585-612.
15. DUAN, D., YUE, Y., YAN, Z., YANG, J., and ENGELHARDT, J.F. (2000). Endosomal processing limits gene transfer to polarized airway epithelia by adeno-associated virus. J. Clin. Invest. 105, 1573-1587.
16. ELOIT, M., GILARDI-HEBENSTREIT, P., TOMA, B., and PERRICAUDET, M. (1990). Construction of a defective adenovirus vector expressing the pseudorabies virus glycoprotein gp50 and its use as a live vaccine. J. Gen. Virol. 71, 2425-2431.
17. EMING, S.A., KRIEG, T., and DAVIDSON, J.M. (2004). Gene transfer in tissue repair: Status, challenges and future directions. Expert Opin. Biol. Ther. 4, 1373-1386.
18. FISHER, K.J., GAO, G.P., WEITZMAN, M.D., DEMATTEO, R., BURDA, J.F., and WILSON, J.M. (1996). Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis. J. Virol. 70, 520-532.
19. FISHER, K.J., JOOSS, K., ALSTON, J., YANG, Y., HAECKER, S.E., HIGH, K., PATHAK, R., RAPER, S.E., and WILSON, J.M. (1997). Recombinant adeno-associated virus for muscle directed gene therapy. Nat. Med. 3, 306-312.
20. GAO, G.P., ALVIRA, M.R., WANG, L., CALCEDO, R., JOHNSTON, J., and WILSON, J.M. (2002). Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc. Natl. Acad. Sci. U.S.A. 99, 11854-11859.
21. HALBERT, C.L., and MILLER, A.D. (2004). AAV-mediated gene transfer to mouse lungs. Methods Mol. Biol. 246, 201-212.
22. HALBERT, C.L., ALEXANDER, I.E., WOLGAMOT, G.M., and MILLER, A.D. (1995). Adeno-associated virus vectors transduce primary cells much less efficiently than immortalized cells. J. Virol. 69, 1473-1479.
23. HALBERT, C.L., STANDAERT, T.A., AITKEN, M.L., ALEXANDER, I.E., RUSSELL, D.W., and MILLER, A.D. (1997). Transduction by adeno-associated virus vectors in the rabbit airway: Efficiency, persistence, and readministration. J. Virol. 71, 5932-5941.
24. HALBERT, C.L., STANDAERT, T.A., WILSON, C.B., and MILLER, A.D. (1998). Successful readministration of adeno-associated virus vectors to the mouse lung requires transient immunosuppression during the initial exposure. J. Virol. 72, 9795-9805.
25. HALBERT, C.L., ALLEN, J.M., and MILLER, A.D. (2001). Adeno-associated virus type 6 (AAV6) vectors mediate efficient transduction of airway epithelial cells in mouse lungs compared to that of AAV2 vectors. J. Virol. 75, 6615-6624.
26. HAN, R.N., LIU, J., TANSWELL, A.K., and POST, M. (1993). Ontogeny of platelet-derived growth factor receptor in fetal rat lung. Microsc. Res. Tech. 26, 381-388.
27. HERMANNS, M.I., UNGER, R.E., KEHE, K., PETERS, K., and KIRKPATRICK, C.J. (2004). Lung epithelial cell lines in coculture with human pulmonary microvascular endothelial cells: development of an alveolo-capillary barrier in vitro. Lab. Invest. 84, 736-752.
28. HUTTER, R., CARRICK, F.E., VALDIVIEZO, C., WOLINSKY, C., RUDGE, J.S., WIEGAND, S.J., FUSTER, V., BADIMON, J.J., and SAUTER, B.V. (2004). Vascular endothelial growth factor regulates reendothelialization and neointima formation in a mouse model of arterial injury. Circulation 110, 2430-2435.
29. JOBE, A.J. (1999). The new BPD: an arrest of lung development. Pediatr. Res. 46, 641-643.
30. KAPLITT, M.G., LEONE, P., SAMULSKI, R.J., XIAO, X., PFAFF, D.W., O'MALLEY, K.L., and DURING, M.J. (1994). Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain. Nat. Genet. 8, 148-154.
31. LAFONT, A., LOIRAND, G., PACAUD, P., VILDE, F., LEMARCHAND, P., and ESCANDE, D. (1997). Vasomotor dysfunction early after exposure of normal rabbit arteries to an adenoviral vector. Hum. Gene Ther. 8, 1033-1040.
32. LI, J., SAMULSKI, R.J., and XIAO, X. (1997). Role for highly regulated rep gene expression in adeno-associated virus vector production. J. Virol. 71, 5236-5243.
33. LOOK, D.C., and BRODY, S.L. (1999). Engineering viral vectors to subvert the airway defense response. Am. J. Respir. Cell Mol. Biol. 20, 1103-1106.
34. MARSZALEK, A., BICZYSKO, W., WASOWICZ, M., DAA, T., and YOKOYAMA, S. (2002). The expression of PDGF, its receptors and capillary morphometry in the developing lung: Quantitative studies. Folia Histochem. Cytobiol. 40, 139-140.
35. MOSS, R.B., RODMAN, D., SPENCER, L.T., AITKEN, M.L., ZEITLIN, P.L., WALTZ, D., MILLA, C., BRODY, A.S., CLANCY, J.P., RAMSEY, B., HAMBLETT, N., and HEALD, A.E. (2004). Repeated adeno-associated virus serotype 2 aerosol-mediated cystic fibrosis transmembrane regulator gene transfer to the lungs of patients with cystic fibrosis: A multicenter, double-blind, placebo-controlled trial. Chest 125, 509-521.
36. MURAMATSU, S., MIZUKAMI, H., YOUNG, N.S., and BROWN, K.E. (1996). Nucleotide sequencing and generation of an infectious clone of adeno-associated virus 3. Virology 221, 208-217.
37. RABINOWITZ, J.E., ROLLING, F., LI, C., CONRATH, H., XIAO, W., XIAO, X., and SAMULSKI, R.J. (2002). Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity. J. Virol. 76, 791-801.
38. RUTLEDGE, E.A., HALBERT, C.L., and RUSSELL, D.W. (1998). Infectious clones and vectors derived from adeno-associated virus (AAV) serotypes other than AAV type 2. J. Virol. 72, 309-319.
39. SCHERLE, W. (1970). A simple method for volumetry of organs in quantitative stereology. Mikroskopie 26, 57-60.
40. SNYDER, R.O., XIAO, X., and SAMULSKI, R.J. (1996). Production of recombinant adeno-associated viral vectors. In: Current Protocols in Human Genetics. J.H.N. Dracopoli, B. Krof, D. Moir, C. Morton, C. Seidman, J. Seidman, and D. Smith, eds. (John Wiley & Sons, New York) pp. 12.11.11-12.11.24.
41. SRIVASTAVA, A. (2002). Obstacles to human hematopoietic stem cell transduction by recombinant adeno-associated virus 2 vectors. J. Cell Biochem. Suppl. 38, 39-45.
42. STRATFORD-PERRICAUDET, L.D., MAKEH, I., PERRICAUDET, M., and BRIAND, P. (1992). Widespread long-term gene transfer to mouse skeletal muscles and heart. J. Clin. Invest. 90, 626-630.
43. TERAMOTO, S., BARTLETT, J.S., MCCARTY, D., XIAO, X., SAMULSKI, R.J., and BOUCHER, R.C. (1998). Factors influencing adeno-associated virus-mediated gene transfer to human cystic fibrosis airway epithelial cells: Comparison with adenovirus vectors. J. Virol. 72, 8904-8912.
44. VIRELLA-LOWELL, I., ZUSMAN, B., FOUST, K., LOILER, S., CONLON, T., SONG, S., CHESNUT, K.A., FERKOL, T., and FLOTTE, T.R. (2005). Enhancing rAAV vector expression in the lung. J. Gene Med. 7, 842-850.
45. WALTER, J., YOU, Q., HAGSTROM, J.N., SANDS, M., and HIGH, K.A. (1996). Successful expression of human factor IX following repeat administration of adenoviral vector in mice. Proc. Natl. Acad. Sci. U.S.A. 93, 3056-3061.
46. WANG, Z., MA, H.I., LI, J., SUN, L., ZHANG, J., and XIAO, X. (2003). Rapid and highly efficient transduction by double-stranded adeno-associated virus vectors in vitro and in vivo. Gene Ther. 10, 2105-2111.
47. WASZAK, P., FRANCO-MONTOYA, M.L., JACOB, M.P., DEPREZ, I., LEVAME, M., LAFUMA, C., HARF, A., and DELACOURT, C. (2002). Effect of intratracheal adenoviral vector administration on lung development in newborn rats. Hum. Gene Ther. 13, 1873-1885.
48. WEIBEL, E.R., and CRUZ-ORIVE, L.M. (1997). Morphometric methods. In: The Lung: Scientific Foundations, 2nd ed. R.G. Crystal and J.B. West, eds. (Raven, Philadelphia, PA) pp. 333-344.
49. XIAO, W., BERTA, S.C., LU, M.M., MOSCIONI, A.D., TAZELAAR, J., and WILSON, J.M. (1998a). Adeno-associated virus as a vector for liver-directed gene therapy. J, Virol. 72, 10222-10226.
50. XIAO, X., LI, J., and SAMULSKI, R.J. (1998b). Production of high-liter recombinant adeno-associated virus vectors in the absence of helper adenovirus. J. Virol. 72, 2224-2232.
51. XIAO, W., CHIRMULE, N., BERTA, S.C., MCCULLOUGH, B., GAO, G., and WILSON, J.M. (1999). Gene therapy vectors based on adeno-associated virus type 1. J. Virol. 73, 3994-4003.
52. XU, Z.L., MIZUGUCHI, H., MAYUMI, T., and HAYAKAWA, T. (2003). Woodchuck hepatitis virus post-transcriptional regulation element enhances transgene expression from adenovirus vectors. Biochim. Biophys. Acta 1621, 266-271.
53. YAN, Z., ZAK, R., ZHANG, Y., DING, W., GODWIN, S., MUNSON, K., PELUSO, R., and ENGELHARDT, J.F. (2004). Distinct classes of proteasome-modulating agents cooperatively augment recombinant adeno-associated virus type 2 and type 5-mediated transduction from the apical surfaces of human airway epithelia. J. Virol. 78, 2863-2874.
54. ZABNER, J., COUTURE, L.A., GREGORY, R.J., GRAHAM, S.M., SMITH, A.E., and WELSH, M.J. (1993). Adenovirus-mediated gene transfer transiently corrects the chloride transport defect in nasal epithelia of patients with cystic fibrosis. Cell 75, 207-216.
55. ZABNER, J., SEILER, M., WALTERS, R., KOTIN, R.M., FULGERAS, W., DAVIDSON, B.L., and CHIORINI, J.A. (2000). Adeno-associated virus type 5 (AAV5) but not AAV2 binds to the apical surfaces of airway epithelia and facilitates gene transfer. J. Virol. 74, 3852-3858.
56. ZEPEDA, M., and WILSON, J.M. (1996). Neonatal cotton rats do not exhibit destructive immune responses to adenoviral vectors. Gene Ther. 3, 973-979.