Biodistribution and Shedding of AAV Vectors

Methods in Molecular Biology

Volumn 807, Issue , 2011, Pages 339-359

  Guiner, Caroline Le ^a   Moullier, Phillipe ^a   Arruda, Valder R ^b  

^a INSERM   (France)

^b UNIVERSITY OF PENNSYLVANIA   (United States)

Author keywords

AAV vector; Biodistribution; DNA extraction; Germ line transmission; Infectious particles detection; PCR analysis; Shedding

Indexed keywords

ADENO ASSOCIATED VIRUS; ANIMAL; ARTICLE; FEMALE; GENE VECTOR; HUMAN; IN VITRO STUDY; MALE; METABOLISM; POLYMERASE CHAIN REACTION; SOUTHERN BLOTTING; SPERM;

ANIMALS; BLOTTING, SOUTHERN; DEPENDOVIRUS; FEMALE; GENETIC VECTORS; HUMANS; MALE; POLYMERASE CHAIN REACTION; SEMEN;

EID: 83455201670     PISSN: 10643745     EISSN: 19406029     Source Type: Book Series    
DOI: 10.1007/978-1-61779-370-7_15     Document Type: Chapter

References (24)

1. EMEA. (2009) General principles to address virus and vector shedding, in ICH Considerations, EMEA/CHMP/ICH/449035/442009.
2. EMEA. (1997) Preclinical safety evaluation of biotechnologicy-derived pharmaceuticals S6, in ICH harmonised tripartite guideline.
3. EMEA. (2009) Addenedum to ICH S6: Preclinical safety evaluation of biotechnologicy-derived pharmaceuticals S6(R1), in ICH draft consensus guideline.
4. Schenk-Braat, E. A., van Mierlo, M. M., Wagemaker, G., Bangma, C. H., and Kaptein, L. C. (2007) An inventory of shedding data from clinical gene therapy trials, J Gene Med 9, 910–921.
5. Stieger, K., Schroeder, J., Provost, N., Mendes-Madeira, A., Belbellaa, B., Le Meur, G., Weber, M., Deschamps, J. Y., Lorenz, B., Moullier, P., and Rolling, F. (2009) Detection of intact rAAV particles up to 6 years after successful gene transfer in the retina of dogs and primates, Mol Ther 17, 516–523.
6. 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.
7. Toromanoff, A., Cherel, Y., Guilbaud, M., Penaud-Budloo, M., Snyder, R. O., Haskins, M. E., Deschamps, J. Y., Guigand, L., Podevin, G., Arruda, V. R., High, K. A., Stedman, H. H., Rolling, F., Anegon, I., Moullier, P., and Le Guiner, C. (2008) Safety and efficacy of regional intravenous (r.i.) versus intramuscular (i.m.) delivery of rAAV1 and rAAV8 to nonhuman primate skeletal muscle, Mol Ther 16, 1291–1299.
8. Ciron, C., Cressant, A., Roux, F., Raoul, S., Cherel, Y., Hantraye, P., Deglon, N., Schwartz, B., Barkats, M., Heard, J. M., Tardieu, M., Moullier, P., and Colle, M. A. (2009) AAV1-, AAV2-and AAV5-Mediated human alpha-Iduronidase Gene Transfer In The Brain of Nonhuman Primate: Vector Diffusion and Bio Distribution, Hum Gene Ther 20, 350–360.
9. Favre, D., Provost, N., Blouin, V., Blancho, G., Cherel, Y., Salvetti, A., and Moullier, P. (2001) Immediate and long-term safety of recombinant adeno-associated virus injection into the nonhuman primate muscle, Mol Ther 4, 559–566.
10. Nathwani, A. C., Rosales, C., McIntosh, J., Rastegarlari, G., Nathwani, D., Raj, D., Nawathe, S., Waddington, S. N., Bronson, R., Jackson, S., Donahue, R. E., High, K. A., Mingozzi, F., Ng, C. Y., Zhou, J., Spence, Y., McCarville, M. B., Valentine, M., Allay, J., Coleman, J., Sleep, S., Gray, J. T., Nienhuis, A. W., and Davidoff, A. M. (2011) Long-term Safety and Efficacy Following Systemic Administration of a Self-complementary AAV Vector Encoding Human FIX Pseudotyped With Serotype 5 and 8 Capsid Proteins, Mol Ther 19, 876–885.
11. EMEA. (2006) General principles to address the risk of inadvertent germline integration of gene therapy vectors, in ICH Considerations, PEMEA/CHMP/ICH/469991/462006.
12. Gonin, P., and Gaillard, C. (2004) Gene transfer vector biodistribution: pivotal safety studies in clinical gene therapy development, Gene Ther 11 Suppl 1, S98–S108.
13. Arruda, V. R., Fields, P. A., Milner, R., Wainwright, L., De Miguel, M. P., Donovan, P. J., Herzog, R. W., Nichols, T. C., Biegel, J. A., Razavi, M., Dake, M., Huff, D., Flake, A. W., Couto, L., Kay, M. A., and High, K. A. (2001) Lack of germline transmission of vector sequences following systemic administration of recombinant AAV-2 vector in males, Mol Ther 4, 586–592.
14. 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.
15. Favaro, P., Downey, H. D., Zhou, J. S., Wright, J. F., Hauck, B., Mingozzi, F., High, K. A., and Arruda, V. R. (2009) Host and vector-dependent effects on the risk of germline transmission of AAV vectors, Mol Ther 17, 1022–1030.
16. Schuettrumpf, J., Liu, J. H., Couto, L. B., Addya, K., Leonard, D. G., Zhen, Z., Sommer, J., and Arruda, V. R. (2006) Inadvertent germline transmission of AAV2 vector: findings in a rabbit model correlate with those in a human clinical trial, Mol Ther 13, 1064–1073.
17. Favaro, P., Finn, J. D., Siner, J. I., Wright, J. F., High, K. A., and Arruda, V. R. (2011) Safety of liver gene transfer following peripheral intravascular delivery of AAV-5 and AAV-6 in a large animal model, Hum Gene Ther 22, 843–852.
18. Salvetti, A., Oreve, S., Chadeuf, G., Favre, D., Cherel, Y., Champion-Arnaud, P., David-Ameline, J., and Moullier, P. (1998) Factors influencing recombinant adeno-associated virus production, Hum Gene Ther 9, 695–706.
19. Zen, Z., Espinoza, Y., Bleu, T., Sommer, J. M., and Wright, J. F. (2004) Infectious titer assay for adeno-associated virus vectors with sensitivity sufficient to detect single infectious events, Hum Gene Ther 15, 709–715.
20. Sharpe, R. (1994) Regulation of spermatogen-esis., in The physiology of reproduction. (Knobil E, N. J., eds., Ed.), pp 1363–1419, Raven Press, Ltd, New York.
21. Penaud-Budloo, M., Le Guiner, C., Nowrouzi, A., Toromanoff, A., Cherel, Y., Chenuaud, P., Schmidt, M., von Kalle, C., Rolling, F., Moullier, P., and Snyder, R. O. (2008) Adeno-associated virus vector genomes persist as episomal chromatin in primate muscle, J Virol 82, 7875–7885.
22. Ni, W., Le Guiner, C., Gernoux, G., Penaud-Budloo, M., Moullier, P., and Snyder, R.O. (2011) Longevity of rAAV vector and plasmid DNA in blood after intramuscular injection in nonhuman primates: implications for gene doping, Gene Ther 18, 709–718.
23. Kazazian, H. H., Jr. (1999) An estimated frequency of endogenous insertional mutations in humans, Nat Genet 22, 130.
24. Goodier, J. L., and Kazazian, H. H., Jr. (2008) Retrotransposons revisited: the restraint and rehabilitation of parasites, Cell 135, 23–35.