Intramuscular administration of AAV overcomes pre-existing neutralizing antibodies in rhesus macaques

Vaccine

Volumn 34, Issue 50, 2016, Pages 6323-6329

  Greig, Jenny A ^a   Calcedo, Roberto ^a   Grant, Rebecca L ^a   Peng, Hui ^a   Medina Jaszek, C Angelica ^a   Ahonkhai, Omua ^a   Qin, Qiuyue ^a   Roy, Soumitra ^a   Tretiakova, Anna P ^a   Wilson, James M ^a  

^a UNIVERSITY OF PENNSYLVANIA   (United States)

Author keywords

AAV; Antibody; Muscle; Vector

Indexed keywords

ADENO ASSOCIATED VIRUS VECTOR; IMMUNOGLOBULIN G; NEUTRALIZING ANTIBODY; VIRUS ANTIBODY;

ANIMAL CELL; ANIMAL EXPERIMENT; ANTIBODY TITER; ARTICLE; CONTROLLED STUDY; GENE EXPRESSION; GENE THERAPY; LIVER; MALE; MOUSE; NONHUMAN; PRIORITY JOURNAL; PROTEIN SECRETION; RHESUS MONKEY; SKELETAL MUSCLE; TRANSGENE; ANIMAL; BLOOD; C57BL MOUSE; DEPENDOPARVOVIRUS; GENE VECTOR; IMMUNOLOGY; INTRAMUSCULAR DRUG ADMINISTRATION; KNOCKOUT MOUSE; PROCEDURES; SEROEPIDEMIOLOGY;

ANIMALS; ANTIBODIES, NEUTRALIZING; ANTIBODIES, VIRAL; DEPENDOVIRUS; GENE EXPRESSION; GENETIC THERAPY; GENETIC VECTORS; INJECTIONS, INTRAMUSCULAR; MACACA MULATTA; MALE; MICE, INBRED C57BL; MICE, KNOCKOUT; SEROEPIDEMIOLOGIC STUDIES; TRANSGENES;

EID: 84998865588     PISSN: 0264410X     EISSN: 18732518     Source Type: Journal    
DOI: 10.1016/j.vaccine.2016.10.053     Document Type: Article

References (20)

1. [1] Gao, G.P., Alvira, M.R., Wang, L., Calcedo, R., Johnston, J., Wilson, J.M., Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc Natl Acad Sci USA 99 (2002), 11854–11859.
2. [2] Calcedo, R., Vandenberghe, L.H., Gao, G., Lin, J., Wilson, J.M., Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses. J Infect Dis 199 (2009), 381–390.
3. [3] Wang, L., Calcedo, R., Bell, P., Lin, J., Grant, R.L., Siegel, D.L., et al. Impact of pre-existing immunity on gene transfer to nonhuman primate liver with adeno-associated virus 8 vectors. Hum Gene Ther 22 (2011), 1389–1401.
4. [4] Flotte, T.R., Trapnell, B.C., Humphries, M., Carey, B., Calcedo, R., Rouhani, F., et al. Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing alpha1-antitrypsin: interim results. Hum Gene Ther 22 (2011), 1239–1247.
5. [5] Brantly, M.L., Chulay, J.D., Wang, L., Mueller, C., Humphries, M., Spencer, L.T., et al. Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy. Proc Natl Acad Sci USA 106 (2009), 16363–16368.
6. [6] Manno, C.S., Chew, A.J., Hutchison, S., Larson, P.J., Herzog, R.W., Arruda, V.R., et al. AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood 101 (2003), 2963–2972.
7. [7] Johnson, P.R., Schnepp, B.C., Zhang, J., Connell, M.J., Greene, S.M., Yuste, E., et al. Vector-mediated gene transfer engenders long-lived neutralizing activity and protection against SIV infection in monkeys. Nat Med 15 (2009), 901–906.
8. [8] Balazs, A.B., Chen, J., Hong, C.M., Rao, D.S., Yang, L., Baltimore, D., Antibody-based protection against HIV infection by vectored immunoprophylaxis. Nature 481 (2012), 81–84.
9. [9] Horwitz, J.A., Halper-Stromberg, A., Mouquet, H., Gitlin, A.D., Tretiakova, A., Eisenreich, T.R., et al. HIV-1 suppression and durable control by combining single broadly neutralizing antibodies and antiretroviral drugs in humanized mice. Proc Natl Acad Sci USA 110 (2013), 16538–16543.
10. [10] Limberis, M.P., Wilson, J.M., Adeno-associated virus serotype 9 vectors transduce murine alveolar and nasal epithelia and can be readministered. Proc Natl Acad Sci USA 103 (2006), 12993–12998.
11. [11] Gao, G., Lu, Y., Calcedo, R., Grant, R.L., Bell, P., Wang, L., et al. Biology of AAV serotype vectors in liver-directed gene transfer to nonhuman primates. Mol Ther 13 (2006), 77–87.
12. [12] Greig, J.A., Peng, H., Ohlstein, J., Medina-Jaszek, C.A., Ahonkhai, O., Mentzinger, A., et al. Intramuscular injection of AAV8 in mice and macaques is associated with substantial hepatic targeting and transgene expression. PLoS ONE, 9, 2014, e112268.
13. [13] Wang, L., Wang, H., Bell, P., McCarter, R.J., He, J., Calcedo, R., et al. Systematic evaluation of AAV vectors for liver directed gene transfer in murine models. Mol Ther 18 (2010), 118–125.
14. [14] Breous, E., Somanathan, S., Wilson, J.M., BALB/c mice show impaired hepatic tolerogenic response following AAV gene transfer to the liver. Mol Ther 18 (2010), 766–774.
15. [15] Wang, B., Li, J., Fu, F.H., Chen, C., Zhu, X., Zhou, L., et al. Construction and analysis of compact muscle-specific promoters for AAV vectors. Gene Ther 15 (2008), 1489–1499.
16. [16] Robinson, J.E., Franco, K., Elliott, D.H., Maher, M.J., Reyna, A., Montefiori, D.C., et al. Quaternary epitope specificities of anti-HIV-1 neutralizing antibodies generated in rhesus macaques infected by the simian/human immunodeficiency virus SHIVSF162P4. J Virol 84 (2010), 3443–3453.
17. [17] Manno, C.S., Pierce, G.F., Arruda, V.R., Glader, B., Ragni, M., Rasko, J.J., et al. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med 12 (2006), 342–347.
18. [18] Wang, L., Calcedo, R., Nichols, T.C., Bellinger, D.A., Dillow, A., Verma, I.M., et al. Sustained correction of disease in naive and AAV2-pretreated hemophilia B dogs: AAV2/8-mediated, liver-directed gene therapy. Blood 105 (2005), 3079–3086.
19. [19] Gardner, M.R., Kattenhorn, L.M., Kondur, H.R., von Schaewen, M., Dorfman, T., Chiang, J.J., et al. AAV-expressed eCD4-Ig provides durable protection from multiple SHIV challenges. Nature 519 (2015), 87–91.
20. [20] Saunders, K.O., Wang, L., Joyce, M.G., Yang, Z.Y., Balazs, A.B., Cheng, C., et al. Broadly neutralizing human immunodeficiency virus type 1 antibody gene transfer protects non-human primates from mucosal simian-human immunodeficiency virus infection. J Virol, 2015.