A preclinical animal model to assess the effect of pre-existing immunity on AAV-mediated gene transfer

Molecular Therapy

Volumn 17, Issue 7, 2009, Pages 1215-1224

  Li, Hua ^a   Lin, Shih Wen ^a,b   Giles Davis, Wynetta ^a   Li, Yan ^a   Zhou, Dongming ^a   Xiang, Zhi Quan ^a   High, Katherine A ^c   Ertl, Hildegund C J ^a  

^a WISTAR INSTITUTE   (United States)

^b UNIVERSITY OF PENNSYLVANIA   (United States)

^c HOWARD HUGHES MEDICAL INSTITUTE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; BLOOD CLOTTING FACTOR 9; CAPSID PROTEIN; GAMMA INTERFERON; INTERLEUKIN 2; NEUTRALIZING ANTIBODY; PARVOVIRUS VECTOR; TUMOR NECROSIS FACTOR;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL TISSUE; ANTIGEN BINDING; ARTICLE; CD8+ T LYMPHOCYTE; CONTROLLED STUDY; CROSS REACTION; CYTOKINE PRODUCTION; DRUG BLOOD LEVEL; GENE EXPRESSION; GENE LOSS; GENE NUMBER; GENE THERAPY; IMMUNIZATION; IMMUNOTHERAPY; IN VITRO STUDY; LIVER CELL; MALE; MOUSE; NATURAL KILLER CELL; NATURAL KILLER T CELL; NONHUMAN; TRANSGENE; VIRAL GENE DELIVERY SYSTEM; VIRUS IMMUNITY;

ADENOVIRIDAE; ANIMALS; B-LYMPHOCYTES; CD8-POSITIVE T-LYMPHOCYTES; CELL LINE; ENZYME-LINKED IMMUNOSORBENT ASSAY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HUMANS; KILLER CELLS, NATURAL; MALE; MICE; MICE, INBRED BALB C; MODELS, ANIMAL; POLYMERASE CHAIN REACTION;

ADENO-ASSOCIATED VIRUS; ADENOVIRIDAE; ANIMALIA; MIRIDAE; MUS;

EID: 67649849935     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2009.79     Document Type: Article

References (27)

1. Manno, CS, Pierce, GF, Arruda, VR, Glader, B, Ragni, M, Rasko, JJ 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.
2. + T-cell responses to adeno-associated virus capsid in humans. Nat Med 13: 419-422.
3. Grimm, D, Zhou, S, Nakai, H, Thomas, CE, Storm, TA, Fuess, S et al. (2003). Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy. Blood 102: 2412-2419.
4. Koeberl, DD, Alexander, IE, Halbert, CL, Russell, DW and Miller, AD (1997). Persistent expression of human clotting factor IX from mouse liver after intravenous injection of adeno-associated virus vectors. Proc Natl Acad Sci USA 94: 1426-1431.
5. Mount, JD, Herzog, RW, Tillson, DM, Goodman, SA, Robinson, N, McCleland, ML et al. (2002). Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy. Blood 99: 2670-2676.
6. Li, C, Hirsch, M, Asokan, A, Zeithaml, B, Ma, H, Kafri, T et al. (2007). Adeno-associated virus type 2 (AAV2) capsid-specific cytotoxic T lymphocytes eliminate only vector-transduced cells coexpressing the AAV2 capsid in vivo. J Virol 81: 7540-7547.
7. + T cells are unable to eliminate AAV-transduced hepatocytes. Mol Ther 15: 792-800.
8. Siders, W, Shields, J, Kaplan, J, Lukason, M, Woodworth, L, Wadsworth, S et al. (2008). Cytotoxic T-Lymphocyte (CTL) responses to the transgene product and not AAV capsid protein limit transgene expression in mice. Hum Gene Ther (epub ahead of print).
9. Wang, L, Figueredo, J, Calcedo, R, Lin, J and Wilson, JM (2007). Cross-presentation of adeno-associated virus serotype 2 capsids activates cytotoxic T cells but does not render hepatocytes effective cytolytic targets. Hum Gene Ther 18: 185-194.
10. + T cells: Implications for their use as vaccines. Blood 110: 1916-1923.
11. + T cell epitopes. Mol Ther 12: 1023-1033.
12. Murphy, SL, Li, H, Zhou, S, Schlachterman, A, High, KA and High, K (2008). Prolonged susceptibility to antibody-mediated neutralization for adeno-associated vectors targeted to the liver. Mol Ther 16: 138-145.
13. Takeda, K, Hayakawa, Y, Van Kaer, L, Matsuda, H, Yagita, H and Okumura, K (2000). Critical contribution of liver natural killer T cells to a murine model of hepatitis. Proc Natl Acad Sci USA 97: 5498-5503.
14. Cardone, M, Polito, VA, Pepe, S, Mann, L, D'Azzo, A, Auricchio, A et al. (2006). Correction of Hunter syndrome in the MPSII mouse model by AAV2/8-mediated gene delivery. Hum Mol Genet 15: 1225-1236.
15. Koeberl, DD, Sun, BD, Damodaran, TV, Brown, T, Millington, DS, Benjamin, DK et al. (2006). Early, sustained efficacy of adeno-associated virus vector-mediated gene therapy in glycogen storage disease type Ia. Gene Ther 13: 1281-1289.
16. McEachern, KA, Nietupski, JB, Chuang, WL, Armentano, D, Johnson, J, Hutto, E et al. (2006). AAV8-mediated expression of glucocerebrosidase ameliorates the storage pathology in the visceral organs of a mouse model of Gaucher disease. J Gene Med 8: 719-729.
17. Moscioni, D, Morizono, H, McCarter, RJ, Stern, A, Cabrera-Luque, J, Hoang, A et al. (2006). Long-term correction of ammonia metabolism and prolonged survival in ornithine transcarbamylase-deficient mice following liver-directed treatment with adeno-associated viral vectors. Mol Ther 14: 25-33.
18. Park, ES, Oh, HJ, Kruger, WD, Jung, SC and Lee, JS (2006). Recombinant adeno-associated virus mediated gene transfer in a mouse model for homocystinuria. Exp Mol Med 38: 652-661.
19. Boonnak, K, Slike, BM, Burgess, TH, Mason, RM, Wu, SJ, Sun, P et al. (2008). Role of dendritic cells in antibody-dependent enhancement of dengue virus infection. J Virol 82: 3939-3951.
20. Mori, S, Takeuchi, T and Kanda, T (2008). Antibody-dependent enhancement of adeno-associated virus infection of human monocytic cell lines. Virology 375: 141-147.
21. Xiang, ZQ, Yang, Y, Wilson, JM and Ertl, HC (1996). A replication-defective human adenovirus recombinant serves as a highly efficacious vaccine carrier. Virology 219: 220-227.
22. Zhou, D, Cun, A, Li, Y, Xiang, Z and Ertl, HC (2006). A chimpanzee-origin adenovirus vector expressing the rabies virus glycoprotein as an oral vaccine against inhalation infection with rabies virus. Mol Ther 14: 662-672.
23. Hangartner, L, Zinkernagel, RM and Hengartner, H (2006). Antiviral antibody responses: The two extremes of a wide spectrum. Nat Rev Immunol 6: 231-243.
24. + T cell functionality and mucosal homing. Virology 367: 156-167.
25. Masopust, D, Ha, SJ, Vezys, V and Ahmed, R (2006). Stimulation history dictates memory CD8 T cell phenotype: Implications for prime-boost vaccination. J Immunol 177: 831-839.
26. Maguire, AM, Simonelli, F, Pierce, EA, Pugh, EN, Mingozzi, F, Bennicelli, J et al. (2008). Safety and efficacy of gene transfer for Leber's congenital amaurosis. N Engl J Med 358: 2240-2248.
27. Hui, DJ, Mingozzi, F, Kleefstra, A, Meulenberg, JM, Edmonson, S, Morin, D et al. (2008). Immunosuppression modulates immune responses to AAV capsid in human subjects undergoing intramuscular gene transfer for lipoprotein lipase deficiency. In 50th ASH Annual Meeting and Exposition, San Francisco, CA, 6-9 Dec 2008.