Production of recombinant adeno-associated viral vectors and use in in vitro and in vivo administration

Current Protocols in Neuroscience

Volumn , Issue SUPPL.57, 2011, Pages

  Gray, Steven J ^a   Choi, Vivian W ^a   Asokan, Aravind ^a   Haberman, Rebecca A ^a   McCown, Thomas J ^a   Samulski, Richard Jude ^a  

^a UNIVERSITY OF NORTH CAROLINA SCHOOL OF MEDICINE   (United States)

Author keywords

Adeno associated virus; Brain; Gene delivery; Gene therapy; Purification; Serotypes; Spinal cord; Transduction; Transfection; Viral vectors

Indexed keywords

GREEN FLUORESCENT PROTEIN; PARVOVIRUS VECTOR;

ARTICLE; BRAIN; DOT HYBRIDIZATION; GENE EXPRESSION; IN VITRO GENE TRANSFER; IN VIVO GENE TRANSFER; LUMBAR PUNCTURE; MICROINJECTION; NONHUMAN; PLASMID; POLYMERASE CHAIN REACTION; PRIORITY JOURNAL; QUANTITATIVE ANALYSIS; SEROTYPE; SPINAL CORD; TRANSGENE; VIRAL GENE DELIVERY SYSTEM; VIRUS PURIFICATION; VIRUS RECOMBINANT;

EID: 84255199681     PISSN: 19348584     EISSN: 19348576     Source Type: Journal    
DOI: 10.1002/0471142301.ns0417s57     Document Type: Article

References (24)

1. Bi, X. and Liu, L.F. 1996. DNA rearrangement mediated by inverted repeats. Proc. Natl. Acad. Sci. U.S.A. 93:819-823.
2. Boissy, R. and Astell, C.R. 1985. An Escherichia coli recBCsbcBrecF host permits the deletionresistant propagation of plasmid clones containing the 5' terminal palindrome of minute virus of mice. Gene 35:179-185.
3. Boussif, O., Lezoualc'h, F., Zanta, M.A., Mergny, M.D., Scherman, D., Demeneix, B., and Behr, J.P. 1995. A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: Polyethylenimine. Proc. Natl. Acad. Sci. U.S.A. 92:7297-7301.
4. Brown, T. 1993. Hybridization analysis of DNA blots. Curr. Protoc. Mol. Biol. 21:2.10.1-2.10.16.
5. Brown, T. 1999a. Southern blotting. Curr. Protoc. Mol. Biol. 21:2.9.1-2.9.15.
6. Brown, T. 1999b. Dot and slot blotting of DNA. Curr. Protoc. Mol. Biol. 21:2.9.15-2.9.20.
7. Burger, C., Gorbatyuk, O.S., Velardo, M.J., Peden, C.S., Williams, P., Zolotukhin, S., Reier, P.J., Mandel, R.J., and Muzyczka, N. 2004. 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.
8. Cukor, G., Blacklow, N.R., Hoggan, D., and Berns, K.I. 1984. Biology of adeno-associated virus. In The Parvoviruses. (K.I. Berns, ed.) pp. 33-66. Plenum, New York.
9. Fairbanks, C.A. 2003. Spinal delivery of analgesics in experimental models of pain and analgesia. Adv. Drug Deliv. Rev. 55:1007-1041.
10. Fu, H., Muenzer, J., Samulski, R.J., Breese, G., Sifford, J., Zeng, X., and McCarty, D.M. 2003. 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.
11. Gray, S.J., Matagne, V, Bachaboina, L, Yadav, S, Ojeda, S, and Samulski, R.J. 2011. Preclinical differences of intravascular AAV9 delivery to neurons and glia: A comparative study of adult mice and non-human primates. Mol. Ther. 19:1058-1069.
12. Heilig, J.S. Karen, L. Elbing, K.L., and Brent, R. 1998. Large-scale preparation of plasmid DNA. Curr. Protoc. Mol. Biol. 41:1.7.1-1.7.16.
13. Hermonat, P.L., Labow, M.A., Wright, R., Berns, K.I., and Muzyczka, N. 1984. Genetics of adenoassociated virus: Isolation and preliminary characterization of adeno-associated virus type 2 mutants. J. Virol. 51:329-339.
14. Mandel, R.J., Rendahl, K.G., Spratt, S.K., Snyder, R.O., Cohen, L.K., and Leff, S.E. 1998. Characterization of intrastriatal recombinant adeno-associated virus-mediated gene transfer of human tyrosine hydroxylase and humanGTPcyclohydrolase I in a rat model of Parkinson's disease. J. Neurosci. 18:4271-4284.
15. McCarty, D.M., Fu, H., Monahan, P.E., Toulson, C.E., Naik, P., and Samulski, R.J. 2003. Adenoassociated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo. Gene Ther. 10:2112-2118.
16. McCown, T.J., Xiao, X., Li, J, Breese, GR, and Samulski, RJ. 1996. Differential and persistent expression patterns of CNS gene transfer by an adeno-associated virus (AAV) vector. Brain Res. 713:99-107.
17. McLaughlin, S.K., Collis, P., Hermonat, P.L., and Muzyczka, N. 1988. Adeno-associated virus general transduction vectors: Analysis of proviral structures. J. Virol. 62:1963-1973.
18. Moore, D., Dowhan, D., Chory, J., and Ribaudo, R.K. 2002. Isolation and purification of large DNA restriction fragments from agarose gels. Curr. Protoc. Mol. Biol. 59:2.6.1-2.6.12.
19. Paxinos, G. and Watson, C. 1986. The Rat Brain in Stereotaxic Coordinates, 2nd ed. Academic Press, London.
20. Rabinowitz, J.E., Rolling, F., Li, C., Conrath, H., Xiao, W., Xiao, X., and Samulski, R.J. 2002. Cross-packaging of a single-adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity. J. Virol. 76:791-801.
21. Samulski, R.J., Chang, L.S., and Shenk, T. 1989. Helper-free stocks of recombinant adenoassociated viruses: Normal integration does not require viral gene expression. J. Virol. 63:3822-3828.
22. Smith, F.E. and McCown, T.J. 1997. AAV vectors: General characteristics and potential use in the central nervous system. In Gene Transfer and Therapy for Neurological Disorders. (E.A. Chiocca and X.O. Breakefield, eds.) pp. 79-88. Humana Press, Totowa, N.J.
23. Struhl, K. 1987. Subcloning of DNA fragments. Curr. Protoc. Mol. Biol. 13:3.16.1-3.16.2.
24. Voytas, D., and Ning Ke, N. 1999. Detection and quantitation of radiolabeled proteins and DNA in gels and blots. Curr. Protoc. Mol. Biol. 48:A.3A.1-A.3A.10.