Recombinant adeno-associated virus

Song, S., M. Scott-Jorgensen, I Wang, A. Poirier, I Crawford, M. Campbell-Thompson, and T. Flotte, Intramuscular administration of recombinant adeno-associated virus 2 alpha-I antitrypsin (rAAV-SERPINAl) vectors in a nonhuman primate model safety and immunologic aspects. Mol Ther, 2002. 6(3) 329. 

Malik, P., McQuiston, S.A., Yu, X.J., et al. (1997). Recombinant adeno-associated virus Med.iates a high level of gene transfer but less efficient integration in the K562 human hematopoietic cell line. J. Virol., 71, 1776-1783. 

Davidson, B.L. (2000). Recombinant adeno-associated virus type 2,4, and 5 vectors transduction of variant cell types and regions in the mammalian central nervous system. Proc. Natl. Acad. Sci. U.S.A., 97, 3428-3432. 

Tsai TH, Chen SL, Xiao X, Chiang YH, et al. 2006. Gene therapy of focal cerebral ischemia using defective recombinant adeno-associated virus vectors. Front Biosci. 11 2061-2070. 

Inoue, T., Yamaza, H., Sakai, Y., Mizuno, S., Ohno, M., Hamasaki, N. et al. (1999) Position-independent human beta-globin gene expression mediated by a recombinant adeno-associated virus vector carrying the chicken beta-globin insulator. J. Hum. Genet., 44, 152-162. 

Duan, D., Yue, Y., Yan, Z. and Engelhardt, J. F. (2000). A new dual-vector approach to enhance recombinant adeno-associated virus-mediated gene expression through intermolecular cis activation. Nat. Med. 6, 559-598. 

Fisher, K. J., Gao, G. P., Weitzman, M. D., De Matteo, R., Burda, J. F. and Wilson, J. M. (1996). Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis. J. Virol. 70, 520-532. 

Flannery, J. G., Zolotukhin, S., Vaquero, M. I., LaVail, M. M., Muzyczka, N. and Hauswirth, W. W. (1997). Efficient photoreceptor-targeted gene expression in vivo by recombinant adeno-associated virus. Proc. Natl. Acad. Sci. USA 94, 6916-6921. 

Kearns, W. G., Afione, S. A., Fulmer, S. B., Pang, M. C., Erikson, D., Egan, M., Landrum, M. J., Flotte, T. R. and Cutting, G. R. (1996). Recombinant adeno-associated virus (AAV-CFTR) vectors do not integrate in a site-specific fashion in an immortalized epithelial cell line. Gene Ther. 3, 748-755. 

Klein, R. L., Meyer, E. M., Peel, A. L., Zolotukhin, S., Meyers, C., Muzyczka, N. and King, M. A. (1998). Neuron-specific transduction in the rat septo-hippocampal or nigrostriatal pathway by recombinant adeno-associated virus vectors. Exp. Neurol. 150, 183-194. 

Miao, C. H., Nakai, H., Thompson, A. R., Storm, T. A., Chiu, W., Snyder, R. O. and Kay, M. A. (2000). Nonrandom transduction of recombinant adeno-associated virus vectors in mouse hepatocytes in vivo Cell cycling does not influence hepatocyte transduction. J. Virol. 74, 3793-3803. 

Murphy, J. E., Zhou, S., Giese, K., Williams, L. T., Escobedo, J. A. and Dwarki, V. J. (1997). Long-term correction of obesity and diabetes in genetically obese mice by a single intramuscular injection of recombinant adeno-associated virus encoding mouse leptin. Proc. Natl. Acad. Sci. USA 94, 13921-13926. 

Song, S., Laipis, P., Embury, J., Berns, K., Crawford, J. and Flotte, T. R. (2001b). Stable therapeutic serum levels of human alpha-1 antitrypsin (AAT) after portal vein injection of recombinant adeno-associated virus (rAAV) vectors. Gene Ther. 8, 1299-1306. 

Xiao, X., Li, J. and Samulski, R. J. (1998). Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus. 

K., Summerford, C., Samulski, R. J. and Muzyczka, N. (1999). Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield. Gene Ther. 6, 973-985. 

Chadeuf, G. et al. (2000). Efficient recombinant adeno-associated virus production by a stable rep-cap HeLa cell line correlates with adenovirus-induced amplification of the integrated rep-cap genome. J. Gene Med. 2, 260-268. 

Clark, K. R., Liu, X., McGrath, J. P. and Johnson, P. R. (1999). Highly purified recombinant adeno-associated virus vectors are biologically active and free of detectable helper and wild-type viruses [In Process Citation]. Hum. Gene Ther. 10, 1031 1039. 

Conway, J. E., Zolotukhin, S., Muzyczka, N., Hayward, G. S. and Byme, B. J. (1997). Recombinant adeno-associated virus type 2 replication and packaging is entirely supported by a herpes simplex vims type 1 amplicon expressing Rep and Cap. J. Virol. 71, 8780-8789. 

Conway, J. E. et al. (1999). High-titer recombinant adeno-associated virus production utilizing a recombinant herpes simplex vims type I vector expressing AAV-2 Rep and Cap. Gene Ther. 6, 986-993. 

Nakai, H. et al. (2001). Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo. J. Virol. 75, 6969-6976. 

Salvetti, A. et al. (1998). Factors influencing recombinant adeno-associated virus production. Hum. Gene Ther. 9, 695-706. 

Samulski, R. J., Chang, L. S. and Shenk, T. (1989). Helper-free stocks of recombinant adeno-associated viruses Normal integration does not require viral gene expression. J. Virol. 63, 3822-3828. 

Schnepp, B. C., Clark, K. R., Klemanski, D. L., Pacak, C. A. and Johnson, P. R. (2003). Genetic fate of recombinant adeno-associated virus vector genomes in muscle. J. Virol. 77, 3495-3504. 

Ponnazhagan, S., Mahendra, G., Kumar, S., Thompson, J. A. and Castillas, M., Jr. (2002). Conjugate-based targeting of recombinant adeno-associated virus type 2 vectors by using avidin-linked ligands. J. Virol. 76, 12900-12907. 

Flotte, T. R., Brantly, M. L., Spencer, L. T., Byrne, B. J., Spencer, C. T., Baker, D. J. and Humphries, M. (2004). Phase I trial of intramuscular injection of a recombinant adeno-associated virus alpha 1-antitrypsin (rAAV2-CB-hAAT) gene vector to AAT-deficient adults. Hum. Gene Ther. 15, 93-128. 

Poirier, A., Campbell-Thompson, M., Tang, Q., Scott-Jorgensen, M., Combee, L., Loiler, S., Crawford, J., Song, S. and Flotte, T. R. (2004). Toxicology and biodistribution studies of a recombinant adeno-associated virus 2-alpha-l antitrypsin vector. Preclinica 2, 43-51. 

Flotte, T., Agarwal, A., Wang, J., Song, S., Fenjves, E. S., Inverardi, L., Chesnut, K, Afione, S., Loiler, S., Wasserfall, C., Kapturczak, M., Ellis, T., Nick, FI. and Atkinson, M. (2001). Efficient ex vivo transduction of pancreatic islet cells with recombinant adeno-associated virus vectors. Diabetes 50, 515-520. 

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