Systemic elimination of de novo capsid protein synthesis from replication-competent AAV contamination in the liver

Human Gene Therapy

Volumn 22, Issue 5, 2011, Pages 625-632

  Lu, Hui ^a   Qu, Guang ^b   Yang, Xiao ^b   Xu, Ruian ^c   Xiao, Weidong ^a  

^a TEMPLE UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

^c HUAQIAO UNIVERSITY   (China)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; CAPSID PROTEIN; MICRORNA;

ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; CYTOMEGALOVIRUS; ENZYME LINKED IMMUNOSORBENT ASSAY; GENE EXPRESSION; GENE TARGETING; GENETIC TRANSDUCTION; GENETIC TRANSFECTION; HUMAN; HUMAN CELL; LIVER; LIVER CELL; MOUSE; NONHUMAN; PROTEIN SYNTHESIS; RECOMBINANT PLASMID; VIRAL CONTAMINATION; VIRUS PARTICLE; VIRUS REPLICATION; WESTERN BLOTTING;

ANALYSIS OF VARIANCE; ANIMALS; BLOTTING, WESTERN; CAPSID PROTEINS; CELL LINE; DEPENDOVIRUS; ENZYME-LINKED IMMUNOSORBENT ASSAY; GALACTOSIDES; GENE EXPRESSION REGULATION, VIRAL; GENE THERAPY; GENETIC VECTORS; HEMOPHILIA B; HUMANS; INDOLES; MICE; MICE, INBRED C57BL; MICRORNAS; OLIGONUCLEOTIDES; TRANSFECTION;

ADENO-ASSOCIATED VIRUS; CYTOMEGALOVIRUS; MIRIDAE;

EID: 79955453097     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/hum.2011.005     Document Type: Article

References (28)

1. Allen, J.M., Debelak, D.J., Reynolds, T.C., and Miller, A.D. (1997). Identification and elimination of replication-competent adeno-associated virus (AAV) that can arise by nonhomolo-gous recombination during AAV vector production. J. Virol. 71, 6816-6822. (Pubitemid 27355342)
2. Brantly, M.L., Chulay, J.D., Wang, L., et al. (2009). Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy. Proc. Natl. Acad. Sci. U.S.A. 106, 16363-16368.
3. Chen, H. (2008). Intron splicing-mediated expression of AAV Rep and Cap genes and production of AAV vectors in insect cells. Mol. Ther. 16, 924-930. (Pubitemid 351587103)
4. Chen, L., Lu, H., Wang, J., et al. (2009). Enhanced factor VIII heavy chain for gene therapy of hemophilia A. Mol. Ther. 17, 417-424.
5. Clément, N., Knop, D.R., and Byrne, B.J. (2009). Large-scale adeno-associated viral vector production using a herpesvirus-based system enables manufacturing for clinical studies. Hum. Gene Ther. 20, 796-806.
6. Ferrari, F.K., Xiao, X., McCarty, D., and Samulski, R.J. (1997). New developments in the generation of Ad-free, high-titer rAAV gene therapy vectors. Nat. Med. 3, 1295-1297.
7. Griffiths-Jones, S. (2004). The microRNA Registry. Nucleic Acids Res. 32, D109-D111. (Pubitemid 38081615)
8. Hauck, B., Murphy, S.L., Smith, P.H., et al. (2009). Undetectable transcription of cap in a clinical AAV vector: implications for preformed capsid in immune responses. Mol. Ther. 17, 144-152.
9. Herzog, R.W., Hagstrom, J.N., Kung, S.H., et al. (1997). Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus. Proc. Natl. Acad. Sci. U.S.A. 94, 5804-5809. (Pubitemid 27241852)
10. Li, C., Goudy, K., Hirsch, M., et al. (2009a). Cellular immune response to cryptic epitopes during therapeutic gene transfer. Proc. Natl. Acad. Sci. U.S.A. 106, 10770-10774.
11. Li, C., Hirsch, M., DiPrimio, N., et al. (2009b). Cytotoxic-T-lymphocyte- mediated elimination of target cells transduced with engineered adeno-associated virus type 2 vector in vivo. J. Virol. 83, 6817-6824.
12. Liu, X., Voulgaropoulou, F., Chen, R., etal. (2000). Selective Rep-Cap gene amplification as a mechanism for high-titer recombinant AAV production from stable cell lines. Mol. Ther. 2, 394-403.
13. Lu, H., Chen, L., Wang, J., et al. (2008). Complete correction of hemophilia A with adeno-associated viral vectors containing a full-size expression cassette. Hum. Gene Ther. 19, 648-654. (Pubitemid 351913523)
14. Maguire, A.M., High, K.A., Auricchio, A., et al. (2009). Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial. Lancet 374, 1597-1605.
15. Manno, C.S., Pierce, G.F., Arruda, V.R., et al. (2006). Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat. Med. 12, 342-347. (Pubitemid 43355084)
16. \+ T cell epitopes. J. Gene Med. 11, 1095-1102.
17. Mays, L.E., Vandenberghe, L.H., Xiao, R., et al. (2009). Adeno-associated virus capsid structure drives CD4-dependent CD8\+ T cell response to vector encoded proteins. J. Immunol. 182, 6051-6060.
18. Mendell, J.R., Campbell, K., Rodino-Klapac, L., et al. (2010). Dystrophin immunity in Duchenne's muscular dystrophy. N. Engl. J. Med. 363, 1429-1437.
19. Mohiuddin, I., Loiler, S., Zolotukhin, I., et al. (2005). Herpesvirus-based infectious titering of recombinant adeno-associated viral vectors. Mol. Ther. 11, 320-326.
20. Murphy, S.L., Bhagwat, A., Edmonson, S., et al. (2008). High-throughput screening and biophysical interrogation of hepa-totropic AAV. Mol. Ther. 16, 1960-1967.
21. Muzyczka, N., and Berns, K.I. (2002). Parvoviridae: the viruses and their replication. In Fields' Virology. D.M. Knipe, P.M. Howley, D.E. Griffin, et al., eds. (Lippincott, Williams & Wilkins, Philadelphia, PA) pp. 2327-2379.
22. Pien, G.C., Basner-Tschakarjan, E., Hui, D.J., et al. (2009). Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors. J. Clin. Invest. 119, 1688-1695.
23. Qiao, C., Li, J., Skold, A., et al. (2002). Feasibility of generating adeno-associated virus packaging cell lines containing induc-ible adenovirus helper genes. J. Virol. 76, 1904-1913. (Pubitemid 34094637)
24. Vandenberghe, L.H., Breous, E., Nam, H.J., et al. (2009a). Naturally occurring singleton residues in AAV capsid impact vector performance and illustrate structural constraints. Gene Ther. 16, 1416-1428.
25. Vandenberghe, L.H., Wilson, J.M., and Gao, G. (2009b). Tailoring the AAV vector capsid for gene therapy. Gene Ther. 16, 311-319.
26. Virag, T., Cecchini, S., and Kotin, R.M. (2009). Producing re-combinant adeno-associated virus in foster cells: overcoming production limitations using a baculovirus-insect cell expression strategy. Hum. Gene Ther. 20, 807-817.
27. Wang, X.S., Khuntirat, B., Qing, K., et al. (1998). Characterization of wild-type adeno-associated virus type 2-like particles generated during recombinant viral vector production and strategies for their elimination. J. Virol. 72, 5472-5480. (Pubitemid 28283329)
28. Wright, J.F., Qu, G., Tang, C., and Sommer, J.M. (2003). Re-combinant adeno-associated virus: formulation challenges and strategies for a gene therapy vector. Curr. Opin. Drug. Discov. Devel. 6, 174-178. (Pubitemid 36368371)