New developments in the generation of Ad-free, high-titer rAAV gene therapy vectors

1. Muzyczka, N. Use of adeno-associated virus as a general transduction vector for mammalian cells. [Review] Curr. Top. Microbiol. Immunol. 158, 97−129 (1992). | PubMed  | ISI | ChemPort |
2. Samulski, R.J. Adeno-associated virus-based vectors for human gene therapy. in Gene Therapy from Laboratory to the Clinic (ed. Hui, K.M.) 232−271 (World Scientific, Singapore, 1994). | ChemPort |
3. Flotte, T.R. et al. Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector. Proc. Natl. Acad. Sci. USA 90, 10613−10617 (1993). | PubMed  | ChemPort |
4. Monahan, P.E. et al. Direct intramuscular injection with recombinant AAV vectors results in sustained expression in a dog model of hemophilia. Gene Ther. (in the press).
5. Kessler, P.D. et al. Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein. Proc. Natl. Acad. Sci. USA 93, 14082−14087 (1996). | Article | PubMed  | ChemPort |
6. Xiao, X. & Samulski, R.J. Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector. J. Virol. 70, 8090−8108 (1996).
7. Kaplitt, M.G. et al. Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain. Nature Genet. 8, 148−154 (1994). | Article | PubMed  | ISI | ChemPort |
8. Snyder, R.O. et al. Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors. Nature Genet. 16, 270−276 (1997). | Article | PubMed  | ISI | ChemPort |
9. Xiao, X., Li, J., McCown, T.J. & Samulski, R.J. Gene transfer by adeno-associated virus vectors into the central nervous system. Exp. Neural. 144, 113−124 (1997). | Article | ChemPort |
10. Samulski, R.J., Chang, L.S. & Shenk, T. A recombinant plasmid from which an infectious adeno-associated virus genome can be excised in vitro and its use to study viral replication. J. Virol. 61, 3096−3101 (1987). | PubMed  | ISI | ChemPort |
11. Samulski, R.J., Chang, L.S. & Shenk, T. Helper-free stocks of recombinant adeno-associated viruses: normal integration does not require viral gene expression. J. Virol. 63, 3822−3828 (1989). | PubMed  | ISI | ChemPort |
12. Clark, K.R., Voulgaropoulou, F., Fraley, D.M. & Johnson, P.R. Cell lines for the production of recombinant adeno-associated virus. Hum. Gene Ther. 6, 1329−1341 (1995). | PubMed  | ISI | ChemPort |
13. Flotte, T.R. et al. An improved system for packaging recombinant adeno-associated virus vectors capable of in vivo transduction. Gene Ther. 2, 29−37 (1995). | PubMed  | ISI | ChemPort |
14. Vincent, K.A., Piraino, S.T. & Wadsworth, S.C. Analysis of recombinant adeno-associated virus packaging and requirements for rep and cap gene products. J. Virol. 71, 1897−1905 (1997). | PubMed  | ISI | ChemPort |
15. Li, J., Samulski, R.J. & Xiao, X. Role for highly regulated rep gene expression in adeno-associated virus vector production. J. Virol. 71, 5236−5243 (1997). | PubMed  | ISI | ChemPort |
16. Carter, B.J. Adeno-associated virus helper functions. in CRC Handbook of Parvoviruses 1 (ed. Tijssen, P.) 255−282 (CRC Press, Boca Raton, Florida, 1990).
17. McCoy, R.D. et al. Pulmonary inflammation induced by incomplete or inactivated adenoviral particles. Hum. Gene Ther. 6, 1553−1560 (1995). | PubMed  | ISI | ChemPort |
18. Ferrari, F.K., Samulski, T., Shenk, T. & Samulski, R.J. Second-strand synthesis is a rate-limiting step for efficient transduction by recombinant adeno-associated virus vectors. J. Virol. 70, 3227−3234 (1996). | PubMed  | ISI | ChemPort |
19. Snyder, R.O., Xiao, X. & Samulski, R.J. Production of recombinant adeno-associated viral vectors. in Current Protocols in Human Genetics (eds. Dracopoli, N. et al.) (John Wiley, New York, 1996).

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