Quantification of high-capacity helper-dependent adenoviral vector genomes in vitro and in vivo, using quantitative TaqMan real-time polymerase chain reaction

Human Gene Therapy

Volumn 17, Issue 5, 2006, Pages 531-544

  Puntel, M ^b   Curtin, J F ^b   Zirger, J M ^b   Muhammad, A K M ^b   Xiong, W ^b   Liu, C ^b   Hu, J ^b   Kroeger, K M ^b   Czer, P ^b   Sciascia, S ^b   Mondkar, S ^b   Lowenstein, P R ^a   Castro, M G ^a  

^a CEDARS SINAI MEDICAL CENTER   (United States)

^b NONE

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; BETA GALACTOSIDASE; CELL DNA; CESIUM CHLORIDE; GENOMIC DNA; SODIUM CHLORIDE; THYMIDINE KINASE; VIRUS DNA;

ACCURACY; ADENOVIRUS; ANIMAL EXPERIMENT; ARTICLE; CLONING; CONTROLLED STUDY; DRUG DISTRIBUTION; DRUG EFFICACY; DRUG MEGADOSE; DRUG SAFETY; FEMALE; GENE NUMBER; HELPER VIRUS; HIGH CAPACITY ADENOVIRUS; HUMAN; HUMAN CELL; IMMUNE SYSTEM; IN VITRO STUDY; IN VIVO STUDY; MOUSE; NONHUMAN; NUCLEOTIDE SEQUENCE; QUANTITATIVE ANALYSIS; RAT; REAL TIME POLYMERASE CHAIN REACTION; REPLICATION COMPETENT ADENOVIRUS; REPRODUCIBILITY; TRANSGENE; VIRUS GENOME;

ADENOVIRIDAE; ANIMALS; BASE SEQUENCE; BIOLOGICAL ASSAY; BRAIN; CELL LINE; DNA, VIRAL; FEMALE; GENETIC VECTORS; GENOME, VIRAL; HELPER VIRUSES; MICE; POLYMERASE CHAIN REACTION;

ADENOVIRIDAE;

EID: 33646921996     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/hum.2006.17.531     Document Type: Article

References (53)

1. BILBAO, R., REAY, D.P., WU, E., ZHENG, H., BIERMANN, V., KOCHANEK, S., and CLEMENS, P.R. (2005). Comparison of high-capacity and first-generation adenoviral vector gene delivery to murine muscle in utero. Gene Ther. 12, 39-47.
2. BOUCHARD, S., MACKENZIE, T.C., RADU, A.P., HAYASHI, S., PERANTEAU, W.H., CHIRMULE, N., and FLAKE, A.W. (2003). Long-term transgene expression in cardiac and skeletal muscle following fetal administration of adenoviral or adeno-associated viral vectors in mice. J. Gene Med. 5, 941-950.
3. BOWERS, W.J., HOWARD, D.F., and FEDEROFF, H.J. (2000). Discordance between expression and genome transfer titering of HSV amplicon vectors: Recommendation for standardized enumeration. Mol. Ther. 1, 294-299.
4. BRUNETTI-PIERRI, N., NICHOLS, T.C., McCORQUODALE, S., MERRICKS, E., PALMER, D.J., BEAUDET, A.L., and NG, P. (2005). Sustained phenotypic correction of canine hemophilia B after systemic administration of helper-dependent adenoviral vector. Hum. Gene Ther. 16, 811-820.
5. CASTRO, M.G., COWEN, R., WILLIAMSON, I.K., DAVID, A., JIMENEZ-DALMARONI, M.J., YUAN, X., BIGLIARI, A., WILLIAMS, J.C., HU, J., and LOWENSTEIN, P.R. (2003). Current and future strategies for the treatment of malignant brain tumors. Pharmacol. Ther. 98, 71-108.
6. COOK, J.L., WALKER, T.A., LEWIS, A.M., JR., RULEY, H.E., GRAHAM, F.L., and PILDER, S.H. (1986). Expression of the adenovirus E1A oncogene during cell transformation is sufficient to induce susceptibility to lysis by host inflammatory cells. Proc. Natl. Acad. Sci. U.S.A. 83, 6965-6969.
7. DION, L.D., FANG, J., and GARVER, R.I., Jr. (1996). Supernatant rescue assay versus polymerase chain reaction for detection of wild type adenovirus-contaminating recombinant adenovirus stocks. J. Virol. Methods 56, 99-107.
8. FITZGERALD, J.C., GAO, G.P., REYES-SANDOVAL, A., PAVLAKIS, G.N., XIANG, Z.Q., WLAZLO, A.P., GILES-DAVIS, W., WILSON, J.M., and ERTL, H.C. (2003). A simian replication-defective adenoviral recombinant vaccine to HIV-1 gag. J. Immunol. 170, 1416-1422.
9. FLINT, J., and SHENK, T. (1997). Viral transactivating proteins. Annu. Rev. Genet. 31, 177-212.
10. GERDES, C.A., CASTRO, M.G., and LOWENSTEIN, P.R. (2000). Strong promoters are the key to highly efficient, noninflammatory and noncytotoxic adenoviral-mediated transgene delivery into the brain in vivo. Mol. Ther. 2, 330-338.
11. GRINES, C., RUBANYI, G.M., KLEIMAN, N.S., MARROTT, P., and WATKINS, M.W. (2003). Angiogenic gene therapy with adenovirus 5 fibroblast growth factor-4 (Ad5FGF-4): A new option for the treatment of coronary artery disease. Am. J. Cardiol. 92, 24N-31N.
12. HARVEY, E.G., LEOPOLD, P.L., HACKETT, N.R., GRASSO, T.M., WILLIAMS, P.M., TUCKER, A.L., KANER, R.J., FERRIS, B., GONDA, L, SWEENEY, T.D., RAMALINGAM, R., KOVESDI, I., SHAK, S., and CRYSTAL, R.G. (1999). Airway epithelial CFTR mRNA expression in cystic fibrosis patients after repetitive administration of a recombinam adenovirus. J. Clin. Invest. 104, 1245-1255.
13. HE, G.A., XUE, G., XIAO, L., WU, J.X., XU, B.L., HUANG, J.L., LIANG, Z.H., XIAO, X., HUANG, B.J., LIU, R.Y., and HUANG, W. (2005). Dynamic distribution and expression in vivo of human endostatin gene delivered by adenoviral vector. Life Sci. 77, 1331-1340.
14. HEDMAN, M., HARTIKAINEN, J., SYVANNE, M., STJERNVALL, J., HEDMAN, A., KIVELA, A., VANNINEN, E., MUSSALO, H., KAUPPILA, E., SIMULA, S., NARVANEN, O., RANTALA, A., PEUHKURINEN, K., NIEMINEN, M.S., LAAKSO, M., and YLA-HERTTUALA, S. (2003). Safety and feasibility of catheter-based local intracoronary vascular endothelial growth factor gene transfer in the prevention of postangioplasty and in-stent restenosis and in the treatment of chronic myocardial ischemia. Phase II results of the Kuopio Angiogenesis Trial (KAT). Circulation 107, 2677-2683.
15. HURTADO-LORENZO, A., MILLAN, E., GONZALEZ-NICOLINI, V., SUWELACK, D., CASTRO, M.G., and LOWENSTEIN, P.R. (2004). Differentiation and transcription factor gene therapy in experimental Parkinson's disease: Sonic hedgehog and Gli-1, but not Nurr-1, protect nigrostriatal cell bodies from 6-OHDA-induced neurodegeneration. Mol. Ther. 10, 507-524.
16. IMMONEN, A., VAPALAHTI, M., TYYNELA, K., HURSKAINEN, H., SANDMAIR, A., VANNINEN, R., LANGFORD, G., MURRAY, N., and YLA-HERTTUALA, S. (2004). AdvHSV-tk gene therapy with intravenous ganciclovir improves survival in human malignant glioma: A randomised, controlled study. Mol. Ther. 10, 967-972.
17. KOCHANEK, S., SCHIEDNER, G., and VOLPERS, C. (2001). High-capacity "gutless" adenoviral vectors. Curr. Opin. Mol. Ther. 3, 454-463.
18. KREPPEL, F., BIERMANN, V., KOCHANEK, S., and SCHIEDNER, G. (2002). A DNA-based method to assay total and infectious particle contents and helper virus contamination in high-capacity adenoviral vector preparations. Hum. Gene Ther. 13, 1151-1156.
19. LOWENSTEIN, P.R., THOMAS, C.E., UMANA, P., GERDES, C.A., VERAKIS, T., BOYER, O., TONDUER, S., KLATZMANN, D. and CASTRO, M.G. (2002). High-capacity, helper-dependent, "gutless" adenovirual vectors for gene transfer into the brain. Methods Enzymol. 346, 292-311.
20. MA, L., BLUYSSEN, H.A., DE RAEYMAEKER, M., LAURYSENS, V., VAN DER BEEK, N., PAVLISKA, H., VAN ZONNEVELD, A.J., TOMME, P., and VAN ES, H.H. (2001). Rapid determination of adenoviral vector titers by quantitative real-time PCR. J. Virol. Methods 93, 181-188.
21. MAIONE, D., DELLA ROCCA, C., GIANNETTI, P., D'ARRIGO, R., LIBERATOSCIOLI, L., FRANLIN, L.L., SANDIG, V., CILIBERTO, G., LA MONICA, N., and SAVING, R. (2001). An improved helper-dependent adenoviral vector allows persistent gene expression after intramuscular delivery and overcomes preexisting immunity to adenovirus. Proc. Natl. Acad. Sci. U.S.A. 98, 5986-5991.
22. MAIZEL, J.V., JR., WHITE, D.O., and SCHARFF, M.D. (1968). The polypeptides of adenovirus. I. Evidence for multiple protein components in the virion and a comparison of types 2, 7A, and 12. Virology 36, 115-125.
23. 1--anti trypsin with negligible toxicity. Hum. Gene Ther. 9, 2709-2716.
24. MORSY, M.A., GU, M., MOTZEL, S., ZHAO, J., LIN, J., SU, Q., ALLEN, H., FRANLIN, L., PARKS, R.J., GRAHAM, F.L., KOCHANEK, S., BETT, A.J., and CASKEY, C.T. (1998). An adenoviral vector deleted for all viral coding sequences results in enhanced safety and extended expression of a leptin transgene. Proc. Natl. Acad. Sci. U.S.A. 95, 7866-7871.
25. NG, P., BEAUCHAMP, C., EVELEGH, C., PARKS, R., and GRAHAM, F.L. (2001). Development of a FLP/frt system for generating helper-dependent adenoviral vectors. Mol. Ther. 3, 809-815.
26. NG, P., EVELEGH, C., CUMMINGS, D., and GRAHAM, F.L. (2002a). Cre levels limit packaging signal excision efficiency in the Cre/loxP helper-dependent adenoviral vector system. J. Virol. 76, 4181-4189.
27. NG, P., PARKS, R.J., and GRAHAM, F.L. (2002b). Preparation of helper-dependent adenoviral vectors. Methods Mol. Med. 69, 371-388.
28. OHASHI, K., NAKAI, H., COUTO, L.B., and KAY, M.A. (2005). Modified infusion procedures affect recombinant adeno-associated virus vector type 2 transduction in the liver. Hum. Gene Ther. 16, 299-306.
29. OHBAYASHI, F., BALAMOTIS, M.A., KISHIMOTO, A., AIZAWA, E., DIAZ, A., HASTY, P., GRAHAM, F.L., CASKEY, C.T., and MITANI, K. (2005). Correction of chromosomal mutation and random integration in embryonic stem cells with helper-dependent adenoviral vectors. Proc. Natl. Acad. Sci. U.S.A. 102, 13628-13633.
30. PALMER, D., and NG, P. (2003). Improved system for helper-dependent adenoviral vector production. Mol. Ther. 8, 846-852.
31. PALMER, D.J., and NG, P. (2004). Physical and infectious titers of helper-dependent adenoviral vectors. A method of direct comparison to the adenovirus reference material. Mol. Ther. 10, 792-798.
32. PARKS, R.J., CHEN, L., ANTON, M., SANKAR, U., RUDNICKI, M.A., and GRAHAM, F.L. (1996). A helper-dependent adenovirus vector system: Removal of helper virus by Cre-mediated excision of the viral packaging signal. Proc. Natl. Acad. Sci. U.S.A. 93, 13565-13570.
33. PEDEN, C.S., BURGER, C., MUZYCZKA, N., and MANDEL, R.J. (2004). Circulating anti-wild-type adeno-associated virus type 2 (AAV2) antibodies inhibit recombinant AAV2 (rAAV2)-mediated, but not rAAVS-mediated, gene transfer in the brain. J. Virol. 78, 6344-6359.
34. RAPER, S.E., CHIRMULE, N., LEE, F.S., WIVEL, N.A., BAGG, A., GAO, G.P., WILSON, J.M., and BATSHAW, M.L. (2003). Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer. Mol. Genet. Metab. 80, 148-158.
35. REDDY, P.S., SAKHUJA, K., GANESH, S., YANG, L., KAYDA, D., BRANN, T., PATTISON, S., GOLIGHTLY, D., IDAMAKANTI, N., PINKSTAFF, A., KALOSS, M., BARJOT, C., CHAMBERLAIN, J.S., KALEKO, M., and CONNELLY, S. (2002). Sustained human factor VIII expression in hemophilia A mice following systemic delivery of a gutless adenoviral vector. Mol. Ther. 5, 63-73.
36. + T cell epitopes. Mol. Ther. 12, 1023-1033.
37. SANDIG, V., YOUIL, R., BETT, A.J., FRANLIN, L.L., OSHIMA, M., MAIONE, D., WANG, F., METZKER, M.L., SAVINO, R., and CASKEY, C.T. (2000). Optimization of the helper-dependent adenovirus system for production and potency in vivo. Proc. Natl. Acad. Sci. U.S.A. 97, 1002-1007.
38. SARAC, M.S., WINDEATT, S., CASTRO, M.G., and LINDBERG, I. (2002). Intrapituitary adenoviral administration of 7B2 can extend life span and reverse endocrinological deficiencies in 7B2 null mice. Endocrinology 143, 2314-2323.
39. SASTRY, L., JOHNSON, T., HOBSON, M.J., SMUCKER, B., and CORNETTA, K. (2002). Titering lentiviral vectors: comparison of DNA, RNA and marker expression methods. Gene Ther. 9, 1155-1162.
40. SCHIEDNER, G., MORRAL, N., PARKS, R.J., WU, Y., KOOPMANS, S.C., LANGSTON, C., GRAHAM, F.L., BEAUDET, A.L., and KOCHANEK, S. (1998). Genomic DNA transfer with a high-capacity adenovirus vector results in improved in vivo gene expression and decreased toxicity. Nat. Genet. 18, 180-183.
41. SCHIEDNER, G., HERTEL, S., JOHNSTON, M., BIERMANN, V., DRIES, V., and KOCHANEK, S. (2002). Variables affecting in vivo performance of high-capacity adenovirus vectors. J. Virol. 76, 1600-1609.
42. SHERING, A.F., BAIN, D., STEWART, K., EPSTEIN, A.L., CASTRO, M.G., WILKINSON, G.W., and LOWENSTEIN, P.R. (1997). Cell type-specific expression in brain cell cultures from a short human cytomegalovirus major immediate early promoter depends on whether it is inserted into herpesvirus or adenovirus vectors. J. Gen. Virol. 78, 445-459.
43. SMITH-ARICA, J.R., MORELLI, A.E., LARREGINA, A.T., SMITH, J., LOWENSTEIN, P.R., and CASTRO, M.G. (2000). Cell-type-specific and regulatable transgenesis in the adult brain: Adenovirus-encoded combined transcriptional targeting and inducible transgene expression. Mol. Ther. 2, 579-587.
44. SOUTHGATE, T.D., KINGSTON, P., and CASTRO, M.G. (2000a). Gene transfer into neural cells in vivo using adenoviral vectors. In: Current Protocols in Neuroscience (Gerfen, C.R., McKay, R., Rogawski, M.A., Sibley, D.R., and Skolnick, P., eds.) (John Wiley & Sons, New York) pp. 4.23.21-24.23.40.
45. SOUTHGATE, T.D., WINDEATT, S., SMITH-ARICA, J., GERDES, C.A., PERONE, MJ., MORRIS, I., DAVIS, J.R., KLATZMANN, D., LOWENSTEIN, P.R., and CASTRO, M.G. (2000b). Transcriptional targeting to anterior pituitary lactotrophic cells using recombinant adenovirus vectors in vitro and in vivo in normal and estrogen/sulpiride-induced hyperplastic anterior pituitaries. Endocrinology 141, 3493-3505.
46. THOMAS, C.E., SCHIEDNER, G., KOCHANEK, S., CASTRO, M.G., and LOWENSTEIN, P.R. (2000). Peripheral infection with adenovirus causes unexpected long-term brain inflammation in animals injected intracranially with first-generation, but not with high-capacity, adenovirus vectors: Toward realistic long-term neurological gene therapy for chronic diseases. Proc. Natl. Acad. Sci. U.S.A. 97, 7482-7487.
47. THOMAS, C.E., BIRKETT, D., ANOZIE, I., CASTRO, M.G., and LOWENSTEIN, P.R. (2001a). Acute direct adenoviral vector cytotoxicity and chronic, but not acute, inflammatory responses correlate with decreased vector-mediated transgene expression in the brain. Mol. Ther. 3, 36-46.
48. THOMAS, C.E., SCHIEDNER, G., KOCHANEK, S., CASTRO, M.G., and LOWENSTEIN, P.R. (2001b). Preexisting antiadenoviral immunity is not a barrier to efficient and stable transduction of the brain, mediated by novel high-capacity adenovirus vectors. Hum. Gene Ther. 12, 839-846.
49. THOMAS, C.E., EDWARDS, P., WICKHAM, T.J., CASTRO, M.G., and LOWENSTEIN, P.R. (2002). Adenovirus binding to the cox-sackievirus and adenovirus receptor or integrins is not required to elicit brain inflammation but is necessary to transduce specific neural cell types. J. Virol. 76, 3452-3460.
50. TOIETTA, G., MANE, V.P., NORONA, W.S., FINEGOLD, M.J., NG, P., McDONAGH, A.F., BEAUDET, A.L., and LEE, B. (2005). Life-long elimination of hyperbilirubinemia in the Gunn rat with a single injection of helper-dependent adenoviral vector. Proc. Natl. Acad. Sci. U.S.A. 102, 3930-3935.
51. UMANA, P., GERDES, C.A., STONE, D., DAVIS, J.R., WARD, D., CASTRO, M.G., and LOWENSTEIN, P.R. (2001). Efficient FLPe recombinase enables scalable production of helper-dependent adenoviral vectors with negligible helper-virus contamination. Nat. Biotechnol. 19, 582-585.
52. WINDEATT, S., SOUTHGATE, T.D., DEWEY, R.A., BOLOGNANI, F., PERONE, M.J., LARREGINA, A.T., MALENIAK, T.C., MORRIS, I.D., GOYA, R.G., KLATZMANN, D., LOWENSTEIN, P.R., and CASTRO, M.G. (2000). Adenovirus-mediated herpes simplex virus type-1 thymidine kinase gene therapy suppresses oestrogen-induced pituitary prolactinomas. J. Clin. Endocrinol. Metab. 85, 1296-1305.
53. XIONG, W., GOVERDHANA, S., SCIASCIA, S.A., CANDOLFI, M., ZIRGER, J.M., BARCIA, C., CURTIN, J.F., KING, G.D., JAITA, G., LIU, C., KROEGER, K., AGADJANIAN, H., MEDINAKAUWE, L., PALMER, D., NG, P., LOWENSTEIN, P.R., and CASTRO, M.G. (2006). Regulatable gutless adenovirus vectors sustain inducible transgene expression in the brain in the presence of an immune response against adenoviruses. J. Virol. 80, 27-37.