Production and characterization of adeno-associated viral vectors (original) (raw)
Xiao, X., Li, J. & Samulski, R.J. Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus. J. Virol.72, 2224–2232 (1998). CASPubMedPubMed Central Google Scholar
Grimm, D., Kern, A., Rittner, K. & Kleinschmidt, J.A. Novel tools for production and purification of recombinant adenoassociated virus vectors. Hum. Gene Ther.9, 2745–2760 (1998). ArticleCASPubMed Google Scholar
Collaco, R.F., Cao, X. & Trempe, J.P. A helper virus-free packaging system for recombinant adeno-associated virus vectors. Gene238, 397–405 (1999). ArticleCASPubMed Google Scholar
Matsushita, T. et al. Adeno-associated virus vectors can be efficiently produced without helper virus. Gene Ther.5, 938–945 (1998). ArticleCASPubMed Google Scholar
Salvetti, A. et al. Factors influencing recombinant adeno-associated virus production. Hum. Gene Ther.9, 695–706 (1998). ArticleCASPubMed Google Scholar
Zhang, X. et al. High-titer recombinant adeno-associated virus production from replicating amplicons and herpes vectors deleted for glycoprotein H. Hum. Gene Ther.10, 2527–2537 (1999). ArticleCASPubMed Google Scholar
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). ArticleCASPubMed Google Scholar
Clark, K.R. Recent advances in recombinant adeno-associated virus vector production. Kidney Int.61, 9–15 (2002). Article Google Scholar
Gao, G.P. et al. High-titer adeno-associated viral vectors from a Rep/Cap cell line and hybrid shuttle virus. Hum. Gene Ther.9, 2353–2362 (1998). ArticleCASPubMed Google Scholar
Zhang, X. & Li, C.Y. Generation of recombinant adeno-associated virus vectors by a complete adenovirus-mediated approach. Mol. Ther.3, 787–792 (2001). ArticleCASPubMed Google Scholar
Zhang, H.G. et al. Recombinant adenovirus expressing adeno-associated virus cap and rep proteins supports production of high-titer recombinant adeno-associated virus. Gene Ther.8, 704–712 (2001). ArticleCASPubMed Google Scholar
Conway, J.E. et al. High-titer recombinant adeno-associated virus production utilizing a recombinant herpes simplex virus type I vector expressing AAV-2 Rep and Cap. Gene Ther.6, 986–993 (1999). ArticleCASPubMed Google Scholar
Qiao, C., Wang, B., Zhu, X., Li, J. & Xiao, X. A novel gene expression control system and its use in stable, high-titer 293 cell-based adeno-associated virus packaging cell lines. J. Virol.76, 13015–13027 (2002). ArticleCASPubMedPubMed Central Google Scholar
Inoue, N. & Russell, D.W. Packaging cells based on inducible gene amplification for the production of adeno-associated virus vectors. J. Virol.72, 7024–7031 (1998). CASPubMedPubMed Central Google Scholar
Urabe, M., Ding, C. & Kotin, R.M. Insect cells as a factory to produce adeno-associated virus type 2 vectors. Hum. Gene Ther.13, 1935–1943 (2002). ArticleCASPubMed Google Scholar
Ruffing, M., Zentgraf, H. & Kleinschmidt, J.A. Assembly of viruslike particles by recombinant structural proteins of adeno-associated virus type 2 in insect cells. J. Virol.66, 6922–6930 (1992). CASPubMedPubMed Central Google Scholar
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). CASPubMedPubMed Central Google Scholar
Gao, G., Vandenberghe, L.H. & Wilson, J.M. New recombinant serotypes of AAV vectors. Curr. Gene Ther.5, 285–297 (2005). ArticleCASPubMed Google Scholar
Choi, V.W., McCarty, D.M. & Samulski, R.J. AAV hybrid serotypes: improved vectors for gene delivery. Curr. Gene Ther.5, 299–310 (2005). ArticleCASPubMedPubMed Central Google Scholar
Gao, G. et al. Purification of recombinant adeno-associated virus vectors by column chromatography and its performance in vivo. Hum. Gene Ther.11, 2079–2091 (2000). ArticleCASPubMed Google Scholar
Zolotukhin, S. et al. Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield. Gene Ther.6, 973–985 (1999). ArticleCASPubMed Google Scholar
Summerford, C. & Samulski, R.J. Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions. J. Virol.72, 1438–1445 (1998). CASPubMedPubMed Central Google Scholar
Walters, R.W., Pilewski, J.M., Chiorini, J.A. & Zabner, J. Secreted and transmembrane mucins inhibit gene transfer with AAV4 more efficiently than AAV5. J. Biol. Chem.277, 23709–23713 (2002). ArticleCASPubMed Google Scholar
Rabinowitz, J.E. et al. Cross-dressing the virion: the transcapsidation of adeno-associated virus serotypes functionally defines subgroups. J. Virol.78, 4421–4432 (2004). ArticleCASPubMedPubMed Central Google Scholar
Kaludov, N., Handelman, B. & Chiorini, J.A. Scalable purification of adeno-associated virus type 2, 4, or 5 using ion-exchange chromatography. Hum. Gene Ther.13, 1235–1243 (2002). ArticleCASPubMed Google Scholar
Grieger, J.C. & Samulski, R.J. Packaging capacity of adeno-associated virus serotypes: impact of larger genomes on infectivity and postentry steps. J. Virol.79, 9933–9944 (2005). ArticleCASPubMedPubMed Central Google Scholar
Clark, K.R., Voulgaropoulou, F. & Johnson, P.R. A stable cell line carrying adenovirus-inducible rep and cap genes allows for infectivity titration of adeno-associated virus vectors. Gene Ther.3, 1124–1132 (1996). CASPubMed Google Scholar
Samulski, R.J. & Shenk, T. Adenovirus E1B 55-Mr polypeptide facilitates timely cytoplasmic accumulation of adeno-associated virus mRNAs. J. Virol.62, 206–210 (1988). CASPubMedPubMed Central Google Scholar
McCarty, D.M. et al. Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo. Gene Ther.10, 2112–2118 (2003). ArticleCASPubMed Google Scholar
Zolotukhin, S. Production of recombinant adeno-associated virus vectors. Hum. Gene Ther.16, 551–557 (2005). ArticleCASPubMed Google Scholar
Choi, V.W., Asokan, A., Haberman, R.A., McCown, T.J. & Samulski, R.J. Production of Recombinant Adeno-Associated Viral Vectors and Use in In Vitro and In Vivo Administration (John Wiley and Sons, Winston-Salem, North Carolina, 2006).
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). CASPubMedPubMed Central Google Scholar
Chen, S. et al. Efficient transduction of vascular endothelial cells with recombinant adeno-associated virus serotype 1 and 5 vectors. Hum. Gene Ther.16, 235–247 (2005). ArticleCASPubMed Google Scholar
Chao, H., Monahan, P.E., Liu, Y., Samulski, R.J. & Walsh, C.E. Sustained and complete phenotype correction of hemophilia B mice following intramuscular injection of AAV1 serotype vectors. Mol. Ther.4, 217–222 (2001). ArticleCASPubMed Google Scholar
Hoggan, M.D., Blacklow, N.R. & Rowe, W.P. Studies of small DNA viruses found in various adenovirus preparations: physical, biological, and immunological characteristics. Proc. Natl. Acad. Sci. USA55, 1467–1474 (1966). ArticleCASPubMedPubMed Central Google Scholar
Fu, H. et al. Self-complementary adeno-associated virus serotype 2 vector: global distribution and broad dispersion of AAV-mediated transgene expression in mouse brain. Mol. Ther.8, 911–917 (2003). ArticleCASPubMed Google Scholar
Wang, Z. et al. Rapid and highly efficient transduction by double-stranded adeno-associated virus vectors in vitro and in vivo. Gene Ther.10, 2105–2111 (2003). ArticleCASPubMed Google Scholar
Liu, Y. et al. Specific and efficient transduction of Cochlear inner hair cells with recombinant adeno-associated virus type 3 vector. Mol. Ther.12, 725–733 (2005). ArticleCASPubMed Google Scholar
Parks, W.P., Green, M., Pina, M. & Melnick, J.L. Physicochemical characterization of adeno-associated satellite virus type 4 and its nucleic acid. J. Virol.1, 980–987 (1967). CASPubMedPubMed Central Google Scholar
Chiorini, J.A., Yang, L., Liu, Y., Safer, B. & Kotin, R.M. Cloning of adeno-associated virus type 4 (AAV4) and generation of recombinant AAV4 particles. J. Virol.71, 6823–6833 (1997). CASPubMedPubMed Central Google Scholar
Liu, G., Martins, I.H., Chiorini, J.A. & Davidson, B.L. Adeno-associated virus type 4 (AAV4) targets ependyma and astrocytes in the subventricular zone and RMS. Gene Ther.12, 1503–1508 (2005). ArticleCASPubMed Google Scholar
Bantel-Schaal, U. & zur Hausen, H. Characterization of the DNA of a defective human parvovirus isolated from a genital site. Virology134, 52–63 (1984). ArticleCASPubMed Google Scholar
Chiorini, J.A., Kim, F., Yang, L. & Kotin, R.M. Cloning and characterization of adeno-associated virus type 5. J. Virol.73, 1309–1319 (1999). CASPubMedPubMed Central Google Scholar
Zabner, J. et al. Adeno-associated virus type 5 (AAV5) but not AAV2 binds to the apical surfaces of airway epithelia and facilitates gene transfer. J. Virol.74, 3852–3858 (2000). ArticleCASPubMedPubMed Central Google Scholar
Lotery, A.J. et al. Adeno-associated virus type 5: transduction efficiency and cell-type specificity in the primate retina. Hum. Gene Ther.14, 1663–1671 (2003). ArticleCASPubMed Google Scholar
Burger, C. et al. Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different regions of the central nervous system. Mol. Ther.10, 302–317 (2004). ArticleCASPubMed Google Scholar
Apparailly, F. et al. Adeno-associated virus pseudotype 5 vector improves gene transfer in arthritic joints. Hum. Gene Ther.16, 426–434 (2005). ArticleCASPubMed Google Scholar
Rutledge, E.A., Halbert, C.L. & Russell, D.W. Infectious clones and vectors derived from adeno-associated virus (AAV) serotypes other than AAV type 2. J. Virol.72, 309–319 (1998). CASPubMedPubMed Central Google Scholar
Blankinship, M.J. et al. Efficient transduction of skeletal muscle using vectors based on adeno-associated virus serotype 6. Mol. Ther.10, 671–678 (2004). ArticleCASPubMed Google Scholar
Halbert, C.L., Allen, J.M. & Miller, A.D. Efficient mouse airway transduction following recombination between AAV vectors carrying parts of a larger gene. Nat. Biotechnol.20, 697–701 (2002). ArticleCASPubMed Google Scholar
Gao, G.P. et al. Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc. Natl. Acad. Sci. USA99, 11854–11859 (2002). ArticleCASPubMedPubMed Central Google Scholar
Louboutin, J.P., Wang, L. & Wilson, J.M. Gene transfer into skeletal muscle using novel AAV serotypes. J. Gene Med.7, 442–451 (2005). ArticleCASPubMed Google Scholar
Wang, Z. et al. Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart. Nat. Biotechnol.23, 321–328 (2005). ArticleCASPubMed Google Scholar
Wang, Z. et al. Widespread and stable pancreatic gene transfer by adeno-associated virus vectors via different routes. Diabetes55, 875–884 (2006). ArticleCASPubMed Google Scholar
Pacak, C.A. et al. Recombinant adeno-associated virus serotype 9 leads to preferential cardiac transduction in vivo. Circ. Res.99, E3–E9 (2006). ArticleCASPubMed Google Scholar
Mori, S., Wang, L., Takeuchi, T. & Kanda, T. Two novel adeno-associated viruses from cynomolgus monkey: pseudotyping characterization of capsid protein. Virology330, 375–383 (2004). ArticleCASPubMed Google Scholar
Muramatsu, S., Mizukami, H., Young, N.S. & Rown, K.E. Nucleotide sequencing and generation of an infections clone of adeno-associated virus 3. Virology221, 208–217 (1996). ArticleCASPubMed Google Scholar