Apparently nonspecific enzyme elevations after portal vein delivery of recombinant adeno-associated virus serotype 2 vector in hepatitis C virus-infected chimpanzees

Human Gene Therapy

Volumn 19, Issue 7, 2008, Pages 681-689

  Flotte, Terence R ^a   Goetzmann, Jason ^b   Caridi, James ^c   Paolillo, Joseph ^c   Conlon, Thomas J ^c,d   Potter, Mark ^c,d   Mueller, Christian ^a   Byrne, Barry J ^c,d  

^a UNIVERSITY OF MASSACHUSETTS MEDICAL SCHOOL   (United States)

^b UNIVERSITY OF LOUISIANA AT LAFAYETTE   (United States)

^c UNIVERSITY OF FLORIDA COLLEGE OF MEDICINE   (United States)

^d UNIVERSITY OF FLORIDA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ALANINE AMINOTRANSFERASE; ASPARTATE AMINOTRANSFERASE; CREATINE KINASE; LIVER ENZYME; MUSCLE ENZYME; PARVOVIRUS VECTOR;

ADENO ASSOCIATED VIRUS; ADENO ASSOCIATED VIRUS 2; ALANINE AMINOTRANSFERASE BLOOD LEVEL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; ASPARTATE AMINOTRANSFERASE BLOOD LEVEL; CHIMPANZEE; CONTROLLED STUDY; CREATINE KINASE BLOOD LEVEL; DRUG BLOOD LEVEL; DRUG LIVER LEVEL; DRUG SAFETY; FATTY LIVER; GENE TRANSFER; HEPATITIS C; HEPATITIS C VIRUS; HISTOPATHOLOGY; LIVER BIOPSY; LIVER TOXICITY; NONHUMAN; PORTAL VEIN; SEROTYPE; VIRAL GENE DELIVERY SYSTEM; VIRUS RECOMBINANT;

ALANINE TRANSAMINASE; ALPHA 1-ANTITRYPSIN; ANIMALS; ANTIBODIES; ANTIBODIES, VIRAL; ASPARTATE AMINOTRANSFERASES; CREATININE; DEPENDOVIRUS; FATTY LIVER; GENETIC VECTORS; HEPATITIS C; INJECTIONS, INTRAVENOUS; LIVER; PAN TROGLODYTES; PHOSPHOTRANSFERASES; PORTAL VEIN; RECOMBINATION, GENETIC; SEROTYPING; TREATMENT OUTCOME; UP-REGULATION;

ADENO-ASSOCIATED VIRUS; ANIMALIA; HEPATITIS C VIRUS; PAN;

EID: 47549090971     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/hum.2007.174     Document Type: Article

References (24)

1. 1-antitrypsin (AAT) vector in AAT-deficient adults. Hum. Gene Ther. 17, 1177-1186.
2. Bukh, J. (2004). A critical role for the chimpanzee model in the study of hepatitis C. Hepatology 39, 1469-1475.
3. Dash, S., Kalkeri, G., McClure, H.M., Garry, R.F., Clejan, S., Thung, S.N., and Murthy, K.K. (2001). Transmission of HCV to a chimpanzee using virus particles produced in an RNA-transfected HepG2 cell culture. J. Med. Virol. 65, 276-281.
4. Donsante, A., Vogler, C., Muzyczka, N., Crawford, J.M., Barker, J., Flotte, T., Campbell-Thompson, M., Daly, T., and Sands, M.S. (2001). Observed incidence of tumorigenesis in long-term rodent studies of rAAV vectors. Gene Ther. 8, 1343-1346.
5. Fernandez, J., Taylor, D., Morhardt, D.R., Mihalik, K., Puig, M., Rice, C.M., Feinstone, S.M., and Major, M.E. (2004). Long-term persistence of infection in chimpanzees inoculated with an infectious hepatitis C virus clone is associated with a decrease in the viral amino acid substitution rate and low levels of heterogeneity. J. Virol. 78, 9782-9789.
6. Flotte, T., Carter, B., Conrad, C., Guggino, W., Reynolds, T., Rosenstein, B., Taylor, G., Walden, S., and Wetzel, R. (1996). A phase I study of an adeno-associated virus-CFTR gene vector in adult CF patients with mild lung disease. Hum. Gene Ther. 7, 1145-1159.
7. Flotte, T.R., Zeitlin, P.L., Reynolds, T.C., Heald, A.E., Pedersen, P., Beck, S., Conrad, C.K., Brass-Ernst, L., Humphries, M., Sullivan, K., Wetzel, R., Taylor, G., Carter, B.J., and Guggino, W.B. (2003). Phase I trial of intranasal and endobronchial administration of a recombinant adeno-associated virus serotype 2 (rAAV2)-CFTR vector in adult cystic fibrosis patients: A two-part clinical study. Hum. Gene Ther. 14, 1079-1088.
8. 1-antitrypsin (rAAV2-CB-hAAT) gene vector to AAT-deficient adults. Hum. Gene Ther. 15, 93-128.
9. Flotte, T.R., Schwiebert, E.M., Zeitlin, P.L., Carter, B.J., and Guggino, W.B. (2005). Correlation between DNA transfer, mRNA expression and functional correlation in airway epithelial cells taken from cystic fibrosis (CF) patients after recombinant adeno-associated virus serotype 2 (rAAV) gene therapy. Hum. Gene Ther. 16, 921-925.
10. High, K.A. (2004). Clinical gene transfer studies for hemophilia B. Semin. Thromb. Hemost. 30, 257-267.
11. Jiang, H., Pierce, G.F., Ozelo, M.C., De Paula, E.V., Vargas, J.A., Smith, P., Sommer, J., Luk, A., Manno, C.S., High, K.A., and Arruda, V.R. (2006). Evidence of multiyear factor IX expression by AAV-mediated gene transfer to skeletal muscle in an individual with severe hemophilia B. Mol. Ther. 14, 452-455.
12. Katayama, K., Kumagai, J., Komiya, Y., Mizui, M., Yugi, H., Kishimoto, S., Yamanaka, R., Tamatsukuri, S., Tomoguri, T., Miyakawa, Y., Tanaka, J., and Yoshizawa, H. (2004). Titration of hepatitis C virus in chimpanzees for determining the copy number required for transmission. Intervirology 47, 57-64.
13. Kay, M.A., Manno, C.S., Ragni, M.V., Larson, P.J., Couto, L.B., McClelland, A., Glader, B., Chew, A.J., Tai, S.J., Herzog, R.W., Arruda, V., Johnson, F., Scallan, C., Skarsgard, E., Flake, A.W., and High, K.A. (2000). Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector [see comments]. Nat. Genet. 24, 257-261.
14. Major, M.E., Dahari, H., Mihalik, K., Puig, M., Rice, C.M., Neumann, A.U., and Feinstone, S.M. (2004). Hepatitis C virus kinetics and host responses associated with disease and outcome of infection in chimpanzees. Hepatology 39, 1709-1720.
15. Manno, C.S., Chew, A.J., Hutchison, S., Larson, P.J., Herzog, R.W., Arruda, V.R., Tai, S.J., Ragni, M.V., Thompson, A., Ozelo, M., Couto, L.B., Leonard, D.G., Johnson, F.A., McClelland, A., Scallan, C., Skarsgard, E., Flake, A.W., Kay, M.A., High, K.A., and Glader, B. (2003). AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood 101, 2963-2972.
16. Manno, C.S., Pierce, G.F., Arruda, V.R., Glader, B., Ragni, M., Rasko, J.J., Ozelo, M.C., Hoots, K., Blatt, P., Konkle, B., Dake, M., Kaye, R., Razavi, M., Zajko, A., Zehnder, J., Rustagi, P.K., Nakai, H., Chew, A., Leonard, D., Wright, J.F., Lessard, R.R., Sommer, J.M., Tigges, M., Sabatino, D., Luk, A., Jiang, H., Mingozzi, F., Couto, L., Ertl, H.C., High, K.A., and Kay, M.A. (2006). Successful transduction of liver in hemophilia by AAV-factor IX and limitations imposed by the host immune response. Nat. Med. 12, 342-347.
17. Poduri, C.D. (2003). Hepatitis C virus (HCV): A review molecular biology of the virus, immunodiagnostics, genomic heterogeneity and the role of virus in hepatocellular carcinoma. Indian J. Exp. Biol. 41, 549-562.
18. 1-antitrypsin (AAT) after portal vein injection of recombinant adeno-associated virus (rAAV) vectors. Gene Ther. 8, 1299-1306.
19. 1-antitrypsin (rAAV-SERPINA1) vectors in a nonhuman primate model: Safety and immunologic aspects. Mol. Ther. 6, 329-335.
20. Wagner, J.A., Moran, M.L., Messner, A.H., Daifuku, R., Conrad, C.K., Reynolds, T., Guggino, W.B., Moss, R.B., Carter, B.J., Wine, J.J., Flotte, T.R., and Gardner, P. (1998). A phase I/II study of tgAAV-CF for the treatment of chronic sinusitis in patients with cystic fibrosis. Hum. Gene Ther. 9, 889-909.
21. Wagner, J.A., Messner, A.H., Moran, M.L., Daifuku, R., Kouyama, K., Desch, J.K., Manly, S., Norbash, A.M., Conrad, C.K., Friborg, S., Reynolds, T., Guggino, W.B., Moss, R.B., Carter, B.J., Wine, J.J., Flotte, T.R., and Gardner, P. (1999). Safety and biological efficacy of an adeno-associated virus vector-cystic fibrosis transmembrane conductance regulator (AAV-CFTR) in the cystic fibrosis maxillary sinus. Laryngoscope 109, 266-274.
22. Wagner, J.A., Nepomuceno, I.B., Messner, A.H., Moran, M.L., Batson, E.P., Dimiceli, S., Brown, B.W., Desch, J.K., Norbash, A.M., Conrad, C.K., Guggino, W.B., Flotte, T.R., Wine, J.J., Carter, B.J., Reynolds, T.C., Moss, R.B., and Gardner, P. (2002). A phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF using maxillary sinus delivery in patients with cystic fibrosis with antrostomies. Hum. Gene Ther. 13, 1349-1359.
23. Xu, L., Daly, T., Gao, C., Flotte, T.R., Song, S., Byrne, B.J., Sands, M.S., and Parker Ponder, K. (2001). CMV-β-actin promoter directs higher expression from an adeno-associated viral vector in the liver than the cytomegalovirus or elongation factor 1α promoter and results in therapeutic levels of human factor X in mice. Hum. Gene Ther. 12, 563-573.
24. Zolotukhin, S., Potter, M., Zolotukhin, I., Sakai, Y., Loiler, S., Fraites, T.J., Jr., Chiodo, V.A., Phillipsberg, T., Muzyczka, N., Hauswirth, W.W., Flotte, T.R., Byrne, B.J., and Snyder, R.O. (2002). Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors. Methods 28, 158-167.